Podcasts about Amicus Therapeutics

This program is supported by educational grants from Amicus Therapeutics, Inc. and Chiesi USA Inc.Fabry disease is an inherited lysosomal storage disease caused by mutations in the GLA gene, disrupting the function of the enzyme, α-galactosidase. This results in the accumulation of globotriaosylceramide (GL-3) and its deacylated form, globotriaosylsphingosine (lyso-GL-3), leading to progressive disruption of multiple organ systems. There are currently three treatment options available for Fabry disease, including two enzyme replacement therapies, agalsidase beta and pegunigalsidase alfa, and a chaperone therapy, migalastat. There are also other treatments in development (e.g., gene therapy, other enzyme replacement therapies) and some that are available in other countries (e.g., agalsidase alfa). Due to the small patient population and variability in Fabry disease severity, it is challenging to develop properly powered, placebo-controlled clinical trials. As such, data shared at conferences like WORLDSymposium 2025 are crucial for guiding best practices in this disease area. This program, led by Dr. Eric Wallace, provides a summary of clinically relevant data presented at WORLDSymposium 2025 that can enhance the care of patients with Fabry disease.  Target AudienceThis activity has been designed to meet the educational needs of physicians specializing in neurology, nephrology, cardiology, gastroenterology, ophthalmology, and dermatology. Other members of the care team may also participate.Learning ObjectivesAfter participating in the activity, learners should be better able to: Describe the latest research being presented to better manage individuals with Fabry disease and its clinical relevance.Eric Wallace, MDProfessor of MedicineDepartment of NephrologyUniversity of Alabama Medical SchoolDisclosure StatementAccording to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.Disclosure of relevant financial relationships are as follows:Faculty Educator/PlannerDr. Wallace discloses the following relevant financial relationships with ineligible companies:Advisory Board Consultant: Sanofi-Genzyme, Chiesi, Kyowa Kirin, Sangamo, NateraGrant/Research Support: Sanofi-Genzyme, Chiesi, Uniqure, Idorsia, Amicus Other Planners for this activity have no relevant financial relationships with any ineligible companies.This activity will review off-label or investigational information.The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.Accreditation and Credit DesignationIn support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.PhysiciansAmerican Academy of CME, Inc., designates this enduring material for a maximum of 0.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPsOther members of the care team will receive a certificate of participation.There are no fees to participate in the activity.  Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments. Your certificate will be emailed to you within 30 days.Hardware/Software RequirementsWindows Requirements: • Operating system: Windows XP Service Pack 2 or later • Browser: Internet Explorer 7 or later, Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connectionMacintosh Requirements: • Operating system: Mac OS X v10.3 or later • Browser: Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connectionPrivacyFor more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm  For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ContactFor any questions, please contact: CEServices@academycme.orgCopyright© 2025. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Guest Name: Cuong Do Title: President & CEO Website: https://bioviepharma.com/ Ticker: BIVI Bio: Cuong Do President & Chief Executive Officer​ Cuong Do, MBA, is the President and Chief Executive Officer of BioVie Inc., a clinical-stage company developing innovative drug therapies for the treatment of neurological and neurodegenerative disorders and advanced liver disease. Do is a highly experienced life sciences executive, who has served in various leadership roles at multiple global enterprises and publicly owned companies over the past 35 years. Prior to his appointment with BioVie, Do was President of Samsung Global Strategy Group where he helped to set the strategic direction for the company's diverse business portfolio, including the growth of its biologics businesses. He was previously the Chief Strategy Officer for Merck, a leading US pharmaceutical company on the Fortune 500 list, where he played a key role in defining the company's strategy, including its focus on oncology and creating its leading position with the anti-PD-1 therapy, pembrolizumab (KEYTRUDA®). Do also held leadership roles in the technology space at Lenovo and Tyco Electronics, and was a senior partner at McKinsey & Company, where he spent 17 years helping to build the company's healthcare, high tech and corporate finance practices. Do is an active entrepreneur who founded Callidus Biopharma (a biotech company working in rare diseases that was acquired by Amicus Therapeutics in 2013), Lysodel Therapeutics (a company developing an innovative pharmaceutical product for obesity), and M6P Therapeutics (a company developing gene and enzyme replacement therapies for rare lysosomal storage disorders). He is a founding member of Social Impact Partners, which is a purpose-driven alliance of global experts and young change-makers working to fight against Alzheimer's Disease. Do also serves as a member of multiple advisory boards to companies and academic institutions, including Seneca Therapeutics, Stanford Medicine, The Tuck School of Business at Dartmouth, Fulbright University Vietnam, Ideal Semiconductors, ILiAD Biotechnologies and others. Do is also a passionate philanthropist, particularly in autism and education. He founded Profectum Foundation, which specializes in providing training and education for professionals and parents of children with autism or special needs. He also founded Identifor, Inc. and the Identifor Foundation to help autistic teenagers identify abilities and strengths to make the transition to adulthood. He currently or formerly served on the boards of Celebrate the Children (a school for children with special needs in New Jersey), Autism Speaks and its Executive, Strategy, Science and Family Services committees and Caring for Cambodia (a non-profit working to educate 7,000+ K-12 students in Siem Reap, Cambodia). He holds a BA from Dartmouth College, and an MBA from the Tuck School of Business at Dartmouth. --- Support this podcast: https://podcasters.spotify.com/pod/show/smartmoneycircle/support

Powered by Peerr   In this week's episode Dr Adaugo shares her journey from being a surgeon to successfully transitioning into the pharmaceutical industry.   We discussed:

Jonathan Soong, General Manager of Amicus Therapeutics, gives insights into being at the forefront of rare disease innovations, patient support programs, and the importance of real world evidence for rare diseases. Get full access to NPC Healthbiz Weekly at healthbiz.substack.com/subscribe

This episode (254) features an interview with John Crowley, who shares his journey into the rare disease space. John's involvement started in 1998 when his daughter Megan was diagnosed with Pompe disease, a rare form of muscular dystrophy. Determined to find a cure, John and his family embarked on a journey of research and entrepreneurship, ultimately founding a biotech company that developed a life-saving enzyme therapy for Pompe disease. Despite initial challenges, including financial struggles and personal sacrifices, John's perseverance led to successful treatments for Megan and other patients. Today, Megan and her brother Patrick are thriving adults, actively contributing to society despite their condition. John's commitment to patient-centered care extends beyond his personal journey. As CEO of Amicus Therapeutics and now as CEO of BIO, he continues to advocate for rare disease patients, emphasizing the importance of universal access to treatment and addressing barriers to healthcare. Throughout the interview, John's resilience and optimism shine through, serving as an inspiration for others facing similar challenges. His dedication to making a difference in the lives of patients and his unwavering determination highlight the transformative power of hope and perseverance in the face of adversity. Also in this episode: Sean plans to burn the clothes he wears on the climb up the world's highest road Rare Resilience: Kelly Barendt Thank you notes: Bio News and the FARA Staff Links and resources: John's previous episode: 144 - DD pt2: Do good and...with John Crowley BIO Make a Wish Foundation Visit our segment partner: #RAREis

Our teacher today is John Crowley. It is difficult to appropriately describe John and the Crowley family's story without drawing comparisons to superheroes. Harrison Ford gave it a shot, and the result is Extraordinary Measures, a blockbuster movie he starred in, alongside Brendan Fraser and Kerry Russell. The movie is based on Gita Anand's book The Cure, which tells the story of John and his wife's journey to save two of their children from a then-untreatable disease called Pompe. Thanks to John, that's no longer the case, and now more than 20 years later, both of his children and many others with Pompe are not only still living, but thriving. Since that time, John's gone on to build several successful biotech companies, including Amicus Therapeutics and Novozyme. And just a few days before the release of this class, it was announced that John is now the new CEO of Biotechnology Innovation Organization, or BIO, which is the leading biotech advocacy, education, and policy organization in the world. There's nobody more purpose-built to give us a history lesson on the biotech industry. Please enjoy this class with the incredible John Crowley. For the full show notes, transcript, and links to mentioned content, check out the episode page here. ----- This episode is brought to you by Summus. Summus is a revolutionary health benefits solution, driving superior employee engagement while dramatically lowering your company's enterprise-level healthcare spend. They're completely transforming the world of health benefits by providing employers and their employees in any location, fast access to over 5,100 of the top medical specialists from America's very best medical centers for support across all health questions. If you're looking for a benefit that drives your employee engagement, truly takes care of your people in their most scary and vulnerable times, all while improving your healthcare ROI, visit GoSummus.info/AOI. This episode is brought to you by Hunt Club. Hunt Club unlocks relationships and helps companies grow. Whether it's recruiting your next hire, landing your next big partner, or financing your business, a trusted introduction always works more effectively. Hunt Club's tech-enabled search model leverages the largest community of its kind to refer amazing talent on your behalf. Additionally, its software program, Atlas, helps organize the entire network of a company or fund and assists in streamlining the right introductions. If you're looking for the ideal solution for all of your talent needs, visit HuntClub.com/AOI. ----- Art of Investing is a property of Pine Grove Studios in collaboration with Colossus, LLC. For more episodes of Art of Investing, visit joincolossus.com/episodes.  Editing and post-production work for this episode was provided by The Podcast Consultant (https://thepodcastconsultant.com). Show Notes (00:00:00) Welcome to the Art of Investing (00:05:09) First Question - The Young John Crowley  (00:09:53) An Educational Odyssey - Naval Academy to Harvard (00:12:40) Embracing Challenges and Pivots in Life (00:16:00) The Crowley's Battle with Pompe Disease (00:23:59) The Biotech Industry's Evolution at the Turn of the Century (00:26:02) The Promise and Challenges of Gene Therapy (00:30:54) The Journey of Starting a Biotech Company: From Novozyme to Amicus (00:32:55) The Challenges and Triumphs of Drug Discovery and Commercialization (00:38:12) Advice for Aspiring Biotech Investors (00:42:26) The Ambitious Vision of Amicus (00:48:10) Acquiring New Technologies and Overcoming Financial Hurdles (00:54:19) John's Personal Triumphs Amidst Professional Challenges (01:02:08) Megan Crowley's Journey and Family Pride (01:14:07) The Current State and Future of Biotechnology (01:21:28) The Impact of M&A in Biotech and Pharma (01:29:11) Leadership Lessons from the Frontlines of Biotech (01:34:32) Balancing Life, Duty, and Passion (01:36:09) Reflecting on Legacy and Purpose

Ozlem Goker-Alpan, MD, Founder and President, LDRTC and David G. Warnock, MD. Professor of Medicine (Emeritus) at University of Alabama at Birmingham discuss best practices to identify and treat kidney problems associated with lysosomal disorders.This CME/CE activity describes the pathophysiologies and management options for lysosomal disease patients with kidney problems. This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. To receive credit for this program, go to https://checkrare.com/learning/ Speakers Ozlem Goker-Alpan, MD, Founder and President, LDRTC David G. Warnock, MD. Professor of Medicine (Emeritus)University of Alabama at BirminghamDisclosuresAffinityCE staff, LDRTC staff, CheckRare staff, planners, and reviewers, have no relevant financial interests to disclose. All faculty disclosures are listed below and are included in the beginning of each presentation.Dr. Goker-Alpan is a consultant, a principal investigator and /or on the speaker bureau, or has received grant support, from the following pharmaceutical companies: Actelion, Amicus Therapeutics, Sanofi, Takeda, Pfizer/Protalix.Dr. Warnock has had research support and/or consulting arrangements with Genzyme Corporation (Sanofi), Shire LLC (Takeda), Amicus, Protalix and Chiesi, Zebra Bio, Walking Fish, Hanmi, and Vera Therapeutics.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Learning ObjectivesAt the end of this activity, participants should be able to:Describe the role of the nephrologist in the team approach to careDescribe best practices to monitor kidney function in lysosomal disordersDescribe best practices to treat kidney disorders lysosomal disordersSupport for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics and Chiesi USA.

Ozlem Goker-Alpan, MD of LDRTC and John Bach, MD, Professor of Neurology at Rutgers School of Medicine discuss best practices to manage respiratory complications in persons with lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. To receive credit for this program, visit https://checkrare.com/learning/p-ldrtc2023-webinar3-assessing-monitoring-managing-respiratory-involvement-in-lysosomal-disorders/DisclosuresAffinityCE staff, LDRTC staff, CheckRare staff, planners, and reviewers, have no relevant financial interests to disclose. All faculty disclosures are listed below and are included in the beginning of each presentation.Ozlem Goker-Alpan, MDFounder and President, Lysosomal & Rare Disorders Research & Treatment Center (LDRTC).Dr. Goker-Alpan is a consultant, a principal investigator and /or on the speaker bureau, or has received grant support, from the following pharmaceutical companies: Actelion, Amicus Therapeutics, Sanofi, Takeda, Pfizer/Protalix.John Bach, MDProfessor of Physical Medicine and Rehabilitation, Professor of Neurology, Rutgers New Jersey Medical Center.Dr. Bach has no relevant financial interest to disclose.Mitigation of Relevant Financial Relationships AffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Learning ObjectivesAt the end of this activity, participants should be able to:• Describe the most common LSDs that have pulmonary complications. • Describe best practices to manage pulmonary symptoms in Pompe disease. • Describe best practices to manage pulmonary symptoms in MPSs.• Describe best practices to manage sleep apnea in lysosomal diseases.PhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesContinuing Nursing Education is provided for this program through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center's Commission on Accreditation (ANCC). This activity provides a maximum of 1.25 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic Counselors Category 2 CEUAffinityCE designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credit™. Genetic counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity.Commercial Support Support for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics and Chiesi USA. Participation CostsThere is no cost to participate in this activity.CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.com.Send customer support requests to cds_support+ldrtc@affinityced.com.

On this week's Biotech Hangout, hosts Brad Loncar, Daphne Zohar, Bruce Booth, Josh Schimmer and Yaron Werber cover the state of the XBI after it hit a 52-week low. They also discuss several deals and financings from the week including Eli Lilly acquiring Point Biopharma ($1.4B) and radiopharma more generally and Sanofi paying Teva to collaborate on inflammatory bowel disease treatment (up to $1.5B). Also Biohaven's offering (~$225M), Amicus Therapeutics' financing with Blackstone ($430M), and Orchard Therapeutics acquired by Kyowa Kirin ($478M).  Takeda announces withdrawal of EXKIVITY after its accelerated approval gets pulled, Syndax, ALX Oncology and Roche data and Boehringer Ingelheim launches biosimilar to AbbVie's Humira at an 81% discount. Other topics include Amgen's KRAS inhibitor under FDA review, Exscientia and AI, Ionis Pharmaceuticals' R&D day and Infinity Pharmaceuticals shuts down. *This episode aired on October 6, 2023

Liv Nixon sits down with Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics. Jayne has over 17 years of experience in the world of rare diseases and patient advocacy, and her passion for improving the lives of individuals and families living with rare diseases is truly inspiring. Join Liv and Jayne as they discuss the importance of hope, compassion, and empathy in challenging times, and how a holistic approach can make a difference in people's lives. Don't miss out on this insightful and uplifting conversation. Subscribe to This Girl KAM and stay tuned for more episodes featuring fabulous women in pharma.00:01:18 Jane Gerchkowicz's journey from journalism to patient advocacy.00:07:15 Women faced challenges in corporate communications.00:13:16 Importance of communication in healthcare.00:18:08 Advocacy and education in genetics.00:26:21 Patient advocacy is crucial.00:30:23 Importance of patient-centered approach.00:36:13 Patient advocacy is evolving and expanding.00:42:43 Collaboration is key for advocacy.00:51:04 Ethics of gene patenting.00:54:46 Importance of informed consent.01:00:50 Importance of genetic counseling01:06:41 Importance of patient advocacy.01:14:11 Patient advocacy is essential.01:18:38 Building something that makes a difference.

This CME/CE activity describes the pathophysiologies and management options for lysosomal disease patients with cardiomyopathies. This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE.This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. To receive credit for this program, go to https://checkrare.com/learning/p-ldrtc2022-webinar2-managing-cardiomyopathies-in-lysosomal-disorders/SpeakersOzlem Goker-Alpan, MD, Founder and President, LDRTCJohn Jefferies, MD, Governor, American College Cardiology, Tennessee Chapter President, American Heart Association, Mid-South Chapter Research Member, St. Jude Children's Research Hospital Team Cardiologist, Memphis Grizzlies .Learning ObjectivesAt the end of this activity, participants should be able to:Describe the role of the cardiologist in the team approach to careDescribe best practices to monitor cardiac symptoms in lysosomal disordersDescribe best practices to treat cardiac symptoms in lysosomal disordersSupport for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics and Chiesi USA.

When people were dying from AIDS in the 1980s and 90s, Congress found a way to expedite the drug approval process and saved countless lives. Established in 1992, the Accelerated Approval Program allows the FDA to speed approval of drugs for patients with serious and life-threatening conditions. In this episode we talk with the father of children with a rare disease, a company CEO working to treat rare diseases and two policy experts on Accelerated Approval.

This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. SpeakersOzlem Goker-Alpan, MD Founder and President, Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)Al-Hertani, MD, Director of the BCH Metabolism and Lysosomal Programs, Boston Children's Hospita; lAssociate Professor of Pediatrics, Harvard Medical SchoolLearning ObjectivesAt the end of this activity, participants should be able to:Describe the need for a team approach to careDescribe best practices to build a multidisciplinary team for a new patientDescribe best practices to maintain a multidisciplinary teamSupport for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics, and Chiesi USA.To earn credit for this CME, go to https://checkrare.com/learning/p-building-and-maintaining-a-multidisciplinary-team-for-lysosomal-disorders/

This 30-minute CME program highlights the latest clinical research about Fabry disease, is a rare X-linked lysosomal disorder that results in the cellular buildup of globotriaosylceramide. Characteristic features of Fabry disease include acroparesthesias, angiokeratomas, hypohidrosis, corneal opacity, gastrointestinal problems, tinnitus, and hearing loss. Fabry disease also involves potentially life-threatening complications such as progressive kidney damage, heart attack, and stroke.This CME program, hosted by Staci Kallish, DO, Associate Professor of Clinical Medicine at the University of Pennsylvania Health System Penn Medicine, provides an overview of the latest clinical research presented at WORLDSymposium 2023 focused on Fabry disease. Supported by educational grants from Amicus Therapeutics and Chiesi USA.To watch the video and obtain CME credit, go to https://checkrare.com/learning-center/courses/

When people were dying from AIDS in the 1980s and 90s, Congress found a way to expedite the drug approval process and saved countless lives. Established in 1992, the Accelerated Approval Program allows the FDA to speed approval of drugs for patients with serious and life-threatening conditions. In this episode we talk with the father of children with a rare disease, a company CEO working to treat rare diseases and two policy experts on Accelerated Approval.

John Crowley, Chairman & Former CEO of Amicus Therapeutics, joins the show to discuss the strides the biotech industry has made in therapies for rare genetic diseases and the industry landscape going forward. This interview originally aired as part of Wharton Business Daily's Business of Healthcare special. Hosted on Acast. See acast.com/privacy for more information.

It was on a Friday the 13th in late winter 1997 when John Crowley's life changed forever.  John and his wife Aileen had been noticing concerning symptoms in their infant daughter Megan for several months, and after a few rounds of testing she was diagnosed with a rare form of muscular dystrophy known as Pompe disease.  Doctors told the Crowleys their daughter likely only had a few years to live, an outlook that ultimately sparked John's remarkable efforts to find treatments for Megan as well as her younger brother Patrick, who was also struck with Pompe. His family's amazing journey was the inspiration for the movie Extraordinary Measures starring Brendan Fraser and Harrison Ford.  Join host Shiv Gaglani in this captivating and inspiring conversation with Crowley, now the Executive Chairman at Amicus Therapeutics, to learn about how he and his team are shedding light on some of the rarest diseases in the world, the promise of new technology in genetics, and the need for an Operation Warp Speed to develop rare disease treatments.  As Crowley puts it, “We can beat nature, we think, in the years and decades ahead. We just oftentimes have to beat time.”Mentioned in this episode: https://amicusrx.com/

This week FirstWord Pharma PLUS editors Michael Flanagan, Virginia Li and Simon King discuss confirmation of positive top-line data for AstraZeneca and Daiichi Sankyo's Enhertu in HER2 ‘low' breast cancer, the implications stemming from Amicus Therapeutics' decision to abort a gene-therapy spin out and whether GlaxoSmithKline and Sanofi's long-awaited COVID-19 vaccine has arrived too late to make a meaningful impact on the pandemic.

Ep. 69: John Crowley's life story was made into a major motion picture called Extraordinary Measures, starring Brandon Fraser and Harrison Ford. This podcast offers its own life and business lessons straight from John himself, including the business he created to save his own children's lives that grew into a global biomedical giant. You will learn: (19:00) How to recruit “passionate entrepreneurs.” (20:00) How to lead empathetically. (43:00) Three steps you need to take to turn big dreams into reality. (44:00) Why spreadsheets should be set aside on day one of corporate retreats. Visit https://donyaeger.com/corporate-competitor-podcast/episode-69/ for a free gift and today's show notes!

In this episode we discuss the mission of Amicus therapeutics to tackle and find treatment for rare genetic disorders with Mr J Crowley (CEO and founder of Amicus Therapeutics). He shares his personal journey into biotechnology to find treatment for his children who had pompe disease, the challenges of balancing being a compassionate, visionary leader with building financial health of a biotechnology company.

This week on Meeting Minds we welcome Anthony DeVergillo, the Digital Communications Associate at Amicus Therapeutics.  Anthony is committed to tearing down the barriers that the pandemic has brought into the public view, and helps organizations find solutions to create inclusive virtual events for disabled people. We talk about the future of the events industry, and why hybrid events are necessary to create the best events possible. You don't want to miss this incredible episode of Meeting Minds!To learn more about the work Anthony is doing, click HERE.

ABOUT THE EPISODE In this episode of PodCatalyst, IABC Executive Director Peter Finn sat down with IABC World Conference 2021 presenter Anthony DeVergillo, internal digital communications associate for Amicus Therapeutics. DeVergillo's presentation, “Inclusive Virtual Experiences in a Post-COVID World,” focuses on why virtual aspects should continue even when we return to in-person events. Here, DeVergillo previews this session, which will feature stories and personal experiences that highlight the importance of inclusivity in virtual events. Listen to this episode to hear more about: Why communicators should prioritize virtual inclusivity and organizations that are getting it right The future of online accessibility and inclusion How communicators can help make meaningful change for inclusivity in the virtual space Like what you hear? Don't miss “Inclusive Virtual Experiences in a Post-COVID World,” on 28 June at 3:20–3:40 p.m. PDT (11:20–11:40 p.m. BST, 28 June; 8:20–8:40 a.m. AEST, 29 June), plus many other inspiring sessions during the virtual IABC World Conference 2021, 28–30 June. Register now to secure your spot for this top-tier virtual event, and look forward to connecting with communication professionals from around the globe. || TRANSCRIPT Visit Catalyst for a full episode transcript: https://catalyst.iabc.com/Podcasts/Podcasts-Article/podcatalyst-episode-11-inclusive-virtual-experiences-a-world-conference-preview-with-anthony-devergillo || LINKS *Episode* https://www.iabc.com/ | https://catalyst.iabc.com/ | https://wc.iabc.com/ | https://wc.iabc.com/Conference-Information/Schedule-of-Events/| https://wc.iabc.com/Registration/Attendee-Registration *Social Media* https://twitter.com/iabc | https://www.linkedin.com/company/iabc/ | https://www.facebook.com/IABCWorld| https://www.youtube.com/user/IABClive | https://www.instagram.com/iabcsnaps/ *IABC Websites* https://www.iabc.com/ | https://catalyst.iabc.com/ | https://wc.iabc.com/ --- Send in a voice message: https://anchor.fm/iabc/message

In this episode of Talks at GS, John F. Crowley, chairman and CEO of Amicus Therapeutics discusses his leadership at Amicus Therapeutics, his efforts to develop cutting-edge treatments for rare diseases and disorders, and his perspective as a biotech innovator on the global response to the pandemic.

Greg teaches the capstone course Sustainable Innovation in Healthcare: Managing Continuous Improvements in Developing Products and Services. He is also Chief Medical and Information Officer at PRA Health Sciences, a leading pharmaceutical service, healthcare data and contract research firm. Previously, he was President and Chief Medical Officer at Castle Creek Pharmaceuticals, President of Rare Disease at Moderna Therapeutics, and had leadership roles at McKinsey & Co., Quintiles, Inc., Proteostasis, Amicus Therapeutics, Domain Associates and Medtronic Neurological. He was co-founder of Immunome Therapeutics. Greg has degrees from Harvard Business School, Yale School of Medicine, Columbia University, and Boston College. He trained at the Brigham and Women's, Children's, and Massachusetts General Hospitals. He is on non-profit advisory boards. Greg writes about innovation for Forbes.

John Crowley is the CEO of Amicus Therapeutics.  He is also a funny and compassionate rare disease Dad.  Join us as we learn life lessons and how to start a company - and Sean learns that a million is quite large. Amicus Therapeutics: https://www.amicusrx.com/ Extraordinary Measures (John's story), starring Brendan Fraser and Harrison Ford: https://g.co/kgs/u7uFgw

Part 2 of our Drug Development Series introduces John Crowley, the CEO of Amicus Therapeutics.  He is also a funny and compassionate rare disease Dad.  Join us as we learn life lessons and how to start a company - and Sean learns that a million is quite large. Amicus Therapeutics: https://www.amicusrx.com/ Extraordinary Measures (John's story), starring Brendan Fraser and Harrison Ford: https://g.co/kgs/u7uFgw

We've had the privilege to work with Amicus Therapeutics on multiple occasions and each time we are moved by their commitment and consistent execution of their corporate mission. This virtual patient panel they facilitated is no exception! Three weeks into the company's work-from-home arrangements due to COVID-19, Amicus wanted to continue connecting their team members with each other and the patient communities they work on behalf of. We were honored to moderate the conversation between Naomi (from the UK), Mike (from New Jersey) and David & Karen (from Arizona) for the global Amicus team and their families. The panelists helped us laugh, encouraged our resilience and inspired us to maximize our time as we embrace the rare disease journey and cope in times of uncertainty.

We’ve had the privilege to work with Amicus Therapeutics on multiple occasions and each time we are moved by their commitment and consistent execution of their corporate mission. This virtual patient panel they facilitated is no exception! Three weeks into the company’s work-from-home arrangements due to COVID-19, Amicus wanted to continue connecting their team members with each other and the patient communities they work on behalf of. We were honored to moderate the conversation between Naomi (from the UK), Mike (from New Jersey) and David & Karen (from Arizona) for the global Amicus team and their families. The panelists helped us laugh, encouraged our resilience and inspired us to maximize our time as we embrace the rare disease journey and cope in times of uncertainty. Listen to our previous patient panel with Amicus here: Be Yourself - LIVE with Amicus

Meet Peter Yacobellis, Councilor-At-Large 2020 Candidate for Montclair, New Jersey! Peter Yacobellis has dedicated his life to public service. He served as a search and rescue supply distributor at ground zero after 9/11, and as an official with the administrations of New York Governors David Paterson and Andrew Cuomo, helped pass marriage equality legislation, ran the New York rent stabilization office and directed the Hurricane Sandy Command Center. Peter studied business management and political science at Adelphi University and environmental sustainability at Columbia University, and is the former Chief of Staff for the brand and marketing team at American Express which oversaw the Shop Small and Small Business Saturday programs. Peter is currently the director of human resources and facilities for Amicus Therapeutics, which develops treatments for children living with extremely rare diseases. Peter has been at the forefront of his generation's fight for LGBTQ rights, propelled by his 1998 discharge from US Air Force basic training under the military's “Don't Ask, Don't Tell” policy. In 2009, he helped organize the march on Washington that led to the policy's reversal.   Peter's connection to Montclair dates to 2007, when he worked out of Garden State Equality's headquarters at the Unitarian Universalist Church. He is a member of Bike & Walk Montclair and the Montclair Film Festival and has exhibited his artwork in town. Peter and his fiancé, Benjamin have been proud Montclair homeowners since 2017.

The teenage years are some of the toughest years most of us live through. Aside from the typical peer pressures and desires to fit in, imagine facing middle and high school with a rare disease. That’s exactly what these three girls have done or are doing right now. Meet Caterina, Maya & Annie as we talk about life with a rare disease. From pay phones and boy bands, to navigating school schedules and surgeries, these three girls talked with Kyle & Sean in front of the committed and supportive staff at Amicus Therapeutics. Their authenticity and outlooks on life are inspiring and enriching to anyone who hears them share. In this episode you’ll be encouraged by their unifying message: Be Yourself! We are grateful to Amicus Therapeutics for allowing us to be a part of their global-staff conference, held at the Make-A-Wish Wishing Place in Monroe, NJ. Additionally, we are thankful for and forever touched by the willingness of Caterina, Maya, & Annie to join us on stage for this forum.

The teenage years are some of the toughest years most of us live through. Aside from the typical peer pressures and desires to fit in, imagine facing middle and high school with a rare disease. That's exactly what these three girls have done or are doing right now.  Meet Caterina, Maya & Annie as we talk about life with a rare disease. From pay phones and boy bands, to navigating school schedules and surgeries, these three girls talked with Kyle & Sean in front of the committed and supportive staff at Amicus Therapeutics. Their authenticity and outlooks on life are inspiring and enriching to anyone who hears them share. In this episode you'll be encouraged by their unifying message: Be Yourself! We are grateful to Amicus Therapeutics for allowing us to be a part of their global-staff conference, held at the Make-A-Wish Wishing Place in Monroe, NJ. Additionally, we are thankful for and forever touched by the willingness of Caterina, Maya & Annie to join us on stage for this forum.

Necessity is the Mother of Innovation: The birth of a child, the death of a loved one - There are events in our lives of such significance that they shake the very foundation of our being. In 1998, John Crowley had a promising law career when his family received a shocking medical diagnosis; Pompe disease, a rare genetic and often fatal neuromuscular disorder, would ravage the bodies of John’s two children, leaving them dependent upon breathing and feeding tubes within 12 months’ time. Blind-sided by the cruel twist of fate, John began a race against time. From leaving a stable job to pursue a cure, to launching his biotech company, Amicus Therapeutics, John shares his gut-wrenching and inspiring journey. Our next guest was serving in the United States Marine Corps when he became frustrated with the lack of relocation resources available to military families. Seeing a gap in the market, David Gran, President of MilitaryByOwner Advertising, Inc., launched a website to advertise homes for sale and rent on U.S. military bases. David quickly understood that quality customer service would be paramount to the success of his business, and came up with an inventive strategy to better assist his target audience. Find out why David’s CS dream team is comprised of military spouses. • [00:00:00] Family Blind-sided by Rare Diagnosis • [00:05:51] Origin Story of Amicus Therapeutics • [00:11:30] Never, Never, Never Give Up • [00:18:20] Building a Website in the Early Internet Era • [00:26:52] Know Your Customer, Be Your Customer • [00:33:21] Manage Systems, Not Individuals

Necessity is the Mother of Innovation: The birth of a child, the death of a loved one - There are events in our lives of such significance that they shake the very foundation of our being.  In 1998, John Crowley had a promising law career when his family received a shocking medical diagnosis; Pompe disease, a rare genetic and often fatal neuromuscular disorder, would ravage the bodies of John’s two children, leaving them dependent upon breathing and feeding tubes within 12 months’ time. Blind-sided by the cruel twist of fate, John began a race against time. From leaving a stable job to pursue a cure, to launching his biotech company, Amicus Therapeutics, John shares his gut-wrenching and inspiring journey. Our next guest was serving in the United States Marine Corps when he became frustrated with the lack of relocation resources available to military families. Seeing a gap in the market, David Gran, President of MilitaryByOwner Advertising, Inc., launched a website to advertise homes for sale and rent on U.S. military bases. David quickly understood that quality customer service would be paramount to the success of his business, and came up with an inventive strategy to better assist his target audience. Find out why David’s CS dream team is comprised of military spouses. [00:00:00] Family Blind-sided by Rare Diagnosis [00:05:51] Origin Story of Amicus Therapeutics [00:11:30] Never, Never, Never Give Up [00:18:20] Building a Website in the Early Internet Era [00:26:52] Know Your Customer, Be Your Customer [00:33:21] Manage Systems, Not Individuals

Today in FirstWord:

Trading Block: What's moving the options market? Options Volume for April: OCC announced that total cleared contract volume in April reached 405,853,179 contracts, a 27% increase from April 2017 volume of 320,547,848 contracts and marking the highest April on record. Earnings! Tuesday - The Walt Disney Company Thursday - Nvidia Odd Block: Bullish paper opening in Coty Inc. (COTY). Rolling calls in Marathon Oil Corporation (MRO). Near ATM earnings calls in Amicus Therapeutics, Inc. (FOLD). Strategy Block: Uncle Mike Tosaw discusses when it might be better to use verticals or diagonal/calendars. Around the Block/Economic Reports: May 8: JOLTS May 9: PPI May 10: Jobless Claims, Consumer Price Index, Treasury Budget May 11: Import and Export Prices, Consumer Sentiment

Trading Block: What's moving the options market? Options Volume for April: OCC announced that total cleared contract volume in April reached 405,853,179 contracts, a 27% increase from April 2017 volume of 320,547,848 contracts and marking the highest April on record. Earnings! Tuesday - The Walt Disney Company Thursday - Nvidia Odd Block: Bullish paper opening in Coty Inc. (COTY). Rolling calls in Marathon Oil Corporation (MRO). Near ATM earnings calls in Amicus Therapeutics, Inc. (FOLD). Strategy Block: Uncle Mike Tosaw discusses when it might be better to use verticals or diagonal/calendars. Around the Block/Economic Reports: May 8: JOLTS May 9: PPI May 10: Jobless Claims, Consumer Price Index, Treasury Budget May 11: Import and Export Prices, Consumer Sentiment

Trading Block: VIX is back in the single digits, hitting 9.54 today. AAPL and the iPhone X. The Key Question on Equifax Options Trade Is Who Initiated the Order. Odd Block: A funky fly/stupid in Finish Line Inc. (FINL). Put spread vs. calls in SPDR Euro Stoxx 50 ETF(FEZ). Size collar trade in Amicus Therapeutics, Inc. (FOLD). Strategy Block: The listeners pick the strategy block: Uncle Mike discusses selling premium with a 9 VIX. Mail Block: Listeners take control of the show Comment from Salman Azhar - I've been selling out of money debit diagonals during earning, on both upside and downside. Essentially exploiting vega during volatility crush. I lose some money at times but the overall strategy has been great. Have I been lucky or does this make sense? Comment from Jedi Marcus - Did the EFX executive know about the breach, but before they disclosed it to the public? Around the Block: VIX futures have been consistently correct, VIX cash consistently wrong. What shoe will drop?

Trading Block: VIX is back in the single digits, hitting 9.54 today. AAPL and the iPhone X. The Key Question on Equifax Options Trade Is Who Initiated the Order. Odd Block: A funky fly/stupid in Finish Line Inc. (FINL). Put spread vs. calls in SPDR Euro Stoxx 50 ETF(FEZ). Size collar trade in Amicus Therapeutics, Inc. (FOLD). Strategy Block: The listeners pick the strategy block: Uncle Mike discusses selling premium with a 9 VIX. Mail Block: Listeners take control of the show Comment from Salman Azhar - I've been selling out of money debit diagonals during earning, on both upside and downside. Essentially exploiting vega during volatility crush. I lose some money at times but the overall strategy has been great. Have I been lucky or does this make sense? Comment from Jedi Marcus - Did the EFX executive know about the breach, but before they disclosed it to the public? Around the Block: VIX futures have been consistently correct, VIX cash consistently wrong. What shoe will drop?

Today in FirstWord:

In this episode of Biotalk podcast, Josh Hamermesh, Vice President of Locust Walk interviews Bradley Campbell, President& COO of Amicus Therapeutics. Amicus is very much at the forefront developing and commercializing important therapies to treat a range of devastating rare and orphan diseases. In fact, this has been an eventful and exciting week at Amicus as the company announced the launch of Galafold in the UK. Galafold is the first oral treatment as well as the first precision medicine product for patients with Fabry’s Disease patients.

John F. Crowley is the Chairman and CEO of Amicus Therapeutics, a publicly traded biotechnology company, which he helped to found in 2005 and is now a 300+ person company in 22 countries. John's involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease -- a severe and often fatal neuromuscular disorder. In his drive to find a cure for them, he left his job and became an entrepreneur as the Co-founder, President, and CEO of Novazyme Pharmaceuticals in 2000, a biotech start-up conducting research on a new experimental treatment for Pompe disease (which he credits as ultimately saving his children's lives). In 2001, Novazyme was acquired by Genzyme Corporation for nearly $200 million. John and his family have been profiled on the front page of The Wall Street Journal and are the subjects of a book by Pulitzer prize-winning journalist Geeta Anand, The Cure: How a Father Raised $100 Million -- and Bucked the Medical Establishment -- in a Quest to Save His Children. The major motion picture Extraordinary Measures, starring Brendan Fraser and Harrison Ford, is inspired by the Crowley family journey. John is the author of a personal memoir: Chasing Miracles: The Crowley Family Journey of Strength, Hope, and Joy. John also served as a commissioned intelligence officer in the U.S. Navy Reserve from 2005-2016. He was assigned to the Joint Special Operations Command (JSOC) and is a veteran of the global war on terrorism, with service in Afghanistan. He graduated with a B.S. in Foreign Service from Georgetown University, and earned a J.D. from the University of Notre Dame Law School and an M.B.A. from Harvard. He previously served (2014-2016) as the National Chairman of the Make-A-Wish Foundation of America and is a founding board member of the Global Genes Project. John is a Henry Crown Fellow at The Aspen Institute. This is an incredibly powerful episode. Enjoy! Show notes and links for this episode can be found at www.fourhourworkweek.com/podcast. This podcast is brought to you by iD Commerce + Logistics. I'm asked all the time about how to scale businesses quickly. Rule number one: remove unnecessary bottlenecks. Many businesses can do so by outsourcing inventory management and fulfillment to a company that makes this its primary focus. iD Commerce + Logistics is just such a company. It helps online retailers and entrepreneurs outgrow their competition by handling all types of details -- from inventory to packing and shipping. I depended on iD to handle these types of details when I launched The 4-Hour Chef so I could focus on promoting the book. As a listener of this podcast, you can get up to $10,000 off your start-up fees and costs waived by visiting tim.blog/scale or idcomlog.com/tim. This podcast is also brought to you by Wealthfront. Wealthfront is a massively disruptive (in a good way) set-it-and-forget-it investing service, led by technologists from places like Apple and world-famous investors. It has exploded in popularity in the last two years and now has more than $2.5B under management. In fact, some of my good investor friends in Silicon Valley have millions of their own money in Wealthfront. Why? Because you can get services previously limited to the ultra-wealthy and only pay pennies on the dollar for them, and it's all through smarter software instead of retail locations and bloated sales teams. Check out wealthfront.com/tim, take their risk assessment quiz, which only takes 2-5 minutes, and they'll show you -- for free -- exactly the portfolio they'd put you in. If you want to just take their advice and do it yourself, you can. Or, as I would, you can set it and forget it. Well worth a few minutes: wealthfront.com/tim.***If you enjoy the podcast, would you please consider leaving a short review on Apple Podcasts/iTunes? It takes less than 60 seconds, and it really makes a difference in helping to convince hard-to-get guests. I also love reading the reviews!For show notes and past guests, please visit tim.blog/podcast.Sign up for Tim’s email newsletter (“5-Bullet Friday”) at tim.blog/friday.For transcripts of episodes, go to tim.blog/transcripts.Interested in sponsoring the podcast? Visit tim.blog/sponsor and fill out the form.Discover Tim’s books: tim.blog/books.Follow Tim:Twitter: twitter.com/tferriss Instagram: instagram.com/timferrissFacebook: facebook.com/timferriss YouTube: youtube.com/timferriss

Shares in Juno Therapeutics and Amicus Therapeutics tumble on disappointing news; and one important drug at Pfizer takes a big step forward. 

Today in FirstWord:

(Bloomberg) -- Taking Stock with Kathleen Hays and Pimm Fox. GUEST: HEALTHCARE: John Crowley, CEO of Amicus Therapeutics, on expanding its pipeline of treatments for rare and orphan diseases.

Today in FirstWord:

Today in FirstWord:

Today in FirstWord:

Today in FirstWord:

Option Block 351: Dollar General Insider Loses Millions Trading Block: A quiet day on the street, given the shortened trading week. A stealth rally in Gold. Traders preparing pre Google earnings. Under 400K ADV in VIX today.   Odd Block: Call spreads trade in Dollar General Corp. (DG), size ratio spreads trade in Carnival Corp (CCL), puts sellers trade in Companhia Energetica Minas. (CIG), and small call buyers trade in Amicus Therapeutics, Inc (FOLD).   Xpress Block: Alex discusses premium harvesting in IdeaHub.   Strategy Block: Uncle Mike Tosaw discusses when it's OK to break the rules and to make new ones.   Mail Block: Question from Aviatrix - Hello all-star panel! Great program. I have a question about option open interest. I don't really understand it. I've read some blogs that say open interest can impact a stock's direction during expiration week. Is this just, as Mark would say, the “tin foil hat crowd” or is there something to this? Should I be tracking open interest as an indicator?  Keep the “wit and wisdom” coming!     Around the Block: Be aware - this is a shortened holiday week.

Option Block 351: Dollar General Insider Loses Millions Trading Block: A quiet day on the street, given the shortened trading week. A stealth rally in Gold. Traders preparing pre Google earnings. Under 400K ADV in VIX today.   Odd Block: Call spreads trade in Dollar General Corp. (DG), size ratio spreads trade in Carnival Corp (CCL), puts sellers trade in Companhia Energetica Minas. (CIG), and small call buyers trade in Amicus Therapeutics, Inc (FOLD).   Xpress Block: Alex discusses premium harvesting in IdeaHub.   Strategy Block: Uncle Mike Tosaw discusses when it's OK to break the rules and to make new ones.   Mail Block: Question from Aviatrix - Hello all-star panel! Great program. I have a question about option open interest. I don't really understand it. I've read some blogs that say open interest can impact a stock's direction during expiration week. Is this just, as Mark would say, the “tin foil hat crowd” or is there something to this? Should I be tracking open interest as an indicator?  Keep the “wit and wisdom” coming!     Around the Block: Be aware - this is a shortened holiday week.

Date:12/30/2008Length: 00:07:18Size: 3.34MBThought Leader: John Crowley, President and CEO, Amicus TherapeuticsIn this episode, Mr. Crowley shares his personal journey to find viable treatements for Pompe disease, a condition which two of his children are afflicted. His amazing story is also showcased in the January issue of PharmaVOICE.Play PodcastFor more information on how you can be featured in a podcast, contact Dan Limbach at dlimbach@pharmavoice.com or call him at (847) 594-0157