Podcasts about idelvion

In this episode, Dr. Steven Pipe, a pediatric hematologist out of the University of Michigan who is also the chair of the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) discusses the major takeaways from the International Society of Thrombosis and Hemostasis’ (ISTH) 2017 Congress, which took place this July in Berlin, Germany. Dr. Pipe shares the latest updates on non-factor replacement therapies, gene therapy, and more on Episode 5 of BloodStream Media’s Ask The Expert podcast series! Disclaimer: The content contained within Ask The Expert is for informational purposes only. Please consult your healthcare provider before making any decisions about treatment. Have a bleeding disorders-related question you’d like to submit to Ask The Expert? Excellent! You can email it to mailbag@bloodstreammedia.com (Subject: Ask The Expert Question), post it on the wall of our Facebook Page, Facebook.com/BloodStreamMedia or tweet it to us @BloodStreamInfo or @pjlynch (host)   ISTH Congress 2017 Headlines: BioMarin and Spark: http://www.biopharmadive.com/news/hemophilia-ISTH-spark-alnylam-gene-therapy-biomarin/446849/ Idelvion: https://hemophilianewstoday.com/2017/07/14/hemophilia-b-pediatric-adult-patients-benefit-from-idelvion-studies-show/ Emicizumab: https://www.dddmag.com/article/2017/07/positive-results-emicizumab-phase-iii-studies-hemophilia-inhibitors Nuwiq: http://www.businesswire.com/news/home/20170709005080/en/ISTH-2017-Berlin-Octapharma-presents-exciting-Nuwiq%C2%AE Q&A with R&D chief of Bioverativ: https://www.dddmag.com/article/2017/07/bioverativ-r-d-chief-breaks-down-data-blood-disorder-drug-pipeline Steven Pipe presents small molecule therapies: http://isthcongressdaily.org/novel-therapies-hemophilia-small-molecules-treatment-hemophilia/  

On this episode, Dr. Steven Pipe- pediatric hematologist and Chair of the National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council- provides an update on new and developing treatment products immediately following his attending the International Society on Thrombosis and Hemostasis (ISTH) Congress. Natalie leads a discussion on a recent CDC study that indicates opioid prescriptions are on the decline, Patrick initiates a game based on a new bleeding disorders online platform, and a community member shares her take on platelet disorders. All this and more on Episode 14 of The BloodStream Podcast!  Opioid Prescriptions Fall After 2010 Peak, C.D.C Report Finds https://www.nytimes.com/2017/07/06/health/opioid-painkillers-prescriptions-united-states.html Rare Platlet Disorders: https://stepsforliving.hemophilia.org/basics-of-bleeding-disorders/types-of-bleeding-disorders/rare-platelet-disorders ISTH Congress 2017 Headlines: BioMarin and Spark: http://www.biopharmadive.com/news/hemophilia-ISTH-spark-alnylam-gene-therapy-biomarin/446849/    Idelvion: https://hemophilianewstoday.com/2017/07/14/hemophilia-b-pediatric-adult-patients-benefit-from-idelvion-studies-show/   Emicizumab: https://www.dddmag.com/article/2017/07/positive-results-emicizumab-phase-iii-studies-hemophilia-inhibitors   Nuwiq: http://www.businesswire.com/news/home/20170709005080/en/ISTH-2017-Berlin-Octapharma-presents-exciting-Nuwiq%C2%AE Q&A with R&D chief of Bioverativ: https://www.dddmag.com/article/2017/07/bioverativ-r-d-chief-breaks-down-data-blood-disorder-drug-pipeline   Steven Pipe presents small molecule therapies: http://isthcongressdaily.org/novel-therapies-hemophilia-small-molecules-treatment-hemophilia/   BloodFeed: https://www.bloodfeed.com Powering Through Podcast http://poweringthrough.org/podcast Ask The Expert Podcast http://bloodstreamexpert.com  

In this episode, Patrick reviews some recent headlines in our Trending Topics segment, shares some takeaways from the American Society of Hematology’s 2016 Annual Meeting during our Like, Share, Comment segment, and during a special Sit-Down segment, we hear from with Executive Directors of patient organizations and chapters from around the country on what 2016 was, and what 2017 could be. The good, bad and otherwise. All this and more on Episode 7 of BloodStream. Sponsors for Episode 7: Presenting Sponsor: Stop The Bleeding! (STB!) Link to STB! Website Supporting Sponsor: Entertainment to Affect Change (E2AC) Link to E2AC Website Trending Topics: Hemophilia News Today publishes articles on “Pain and pain management in hemophilia.” Link. DailyMail.com reports on, “How a blood test could save you [UK Women] from infertility.” Link Blood Journal publishes, “Patient Reported Outcomes to Assess Quality of Hemophilia Care in North India.” Link MASAC publishes four new recommendations. Link Like Share Comment:     My Life, Our Future (MLOF) reaches 5,000 patients enrolled. Link MLOF website: Link Video on MLOF: Link American Society of Hematology (ASH) Headlines of Interest: Alnylam Pharmaceuticals presents interim results from study of investigational, subcutaneously-administered fitusiran (for patients with hemophilia A or B who also have inhibitors). Link CSL Behring presents Phase III trial results from IDELVION (long-acting recombinant product for hemophilia B). Link Novo Nordisk presents data from SMART-7 study of NovoSeven. Link Sanagmo BioSciences presents data from SB-525, a gene therapy treatment currently being studied in mice and non-human primates. Link Spark Therapeutics presents preliminary results from investigational, gene therapy agent SPK-9001 intended for the treatment of people with hemophilia B. Link University of Pennsylvania scientists successfully cure mice with hemophilia B using CRISPR. Link Octapharma announces two clinical trials studying how Wilate may be used to treat people with hemophilia A. Link World AIDS Day at the National AIDS Memorial Grove Honors the Hemophilia Community. Video of Powering Through panel featuring NHF CEO Val Bias, HFA CEO Kimberly Haugstad, COTT President Carl Weixler, and The Grove’s ED John Cunningham. Link NHF CEO Val Bias letter regarding HIV/AIDS Memorial. Link   Sit-Down Segment: Jeremy Griffin, from the New York City Hemophilia Chapter Rich Pezzillo, from the New England Hemophilia Chapter Michelle Kim, from the Southern California Hemophilia Chapter Bob Robinson, from the Bleeding Disorders Alliance of Illinois BloodStream on Social: BloodStream Facebook Page BloodStream Twitter Account BloodStream Host Patrick James Lynch on Twitter and Instagram.   Subscribe to BloodStream: iTunes: http://bit.ly/bloodstreamitunes Stitcher: http://bit.ly/bloodstreamstitcher LibSyn: http://bit.ly/bloodstreamlibsyn SoundCloud: http://bit.ly/bloodstreamSC TuneIn: http://bit.ly/bloodstreamtunein Google Play: http://bit.ly/bloodstreamPlay

In this episode, we take a look back at a historic summer that saw the National Hemophilia Foundation's (NHF) Annual Meeting and the World Federation of America's (WFH) World Congress convene in Orlando for the largest gathering ever of people with bleeding disorders. In addition to featuring some conference related content, our Like Share Comment segment highlight a story on Elijah Warren, a high school swimming star with hemophilia as well as a recent article from John Hopkins University on the power of humor in healthcare. Finally, we close this month's episode with a remembrance of Matt Stinger, a community member and advocate who passed unexpectedly this month at 33 years old. Headlines Novo Nordisk’s B-HERO-S study has found, to the surprise of some, that many patients with mild or moderate hemophilia B in the United States experience their condition negatively impacting their career, education, and recreational activities- something that can be overlooked in those affected by mild or moderate hemophilia as opposed to severe. Link. High schooler Federico Parres is one of five team members on the VEX Robotics World Championship Team F, responsible for creating Caerus-7, a ball-flinging robot. Federico lives with severe hemophilia A and plans on becoming an engineer one day. Link. Bayers’ Hemophilia Awards Program recently announced 16 physicians and researchers from 11 countries who will receives grant money totaling about two million dollars, including 6 individuals from the United States. This is the fourteenth year Bayer has run this program. Link A study published in the European journal Haematologica examines a new potential model for how to treatment patients with hemophilia A who undergo surgery. Something I personally find interesting given our ongoing need to learn more about aging with hemophilia. Link The FDA has approved a new needleless reconstitution device called Baxject III for Shire’s long-lasting factor FVIII therapy Adynovate. For those familiar, it is a very similar device to that which is found in Advate. Link Two medical advisory updates have been announced by the National Hemophilia Foundation regarding existing products: NHF Medical Advisory 419 (four-one-nine) states that Bayer has announced a voluntary recall of two lots of Kogenate FS for a loss of potency. The lot numbers are 270TN1C and 270R978. For more information or to arrange for an exchange please call the Recall Processing Center at 855-838-5782. Link And NHF Medical Advisory 420 (four-two-zero) states that CSL Behring has announced a voluntary recall of two lots of Helixate FS for a loss of potency. Those lot numbers are 270TN1G and 270R979. For more information or to arrange an exchange for these products, call CSL Behring’s Customer Service at 1800-683-1288. Link Product Related Headlines (not mentioned in the show) Genentech/Roche has released encouraging Phase 1/2 extension results for their investigational subcutaneous medicine, emicizumab-previously referred to as ACE910. Link The Netherlands-based uniQure, a company self-described as a leader in human gene therapy, announced encouraging clinical data from their ongoing Phase 1/2 trail of AMT-060, an investigational gene therapy for patients with hemophilia B. Link​ BioMarin has presented positive interim data from a Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for people severe hemophilia A. The company has announced intent to begin another study with an eye toward regulatory approval in both the US and Europe. Link CSL Behring’s hemophilia B therapy Idelvion shows promise after encouraging Phase 3 trial results. Link The company also posted positive results from a Phase III study from its FDA approved, hemophilia A treatment AFSTYLA, unique for being the first and only single-chain recombinant product available in the US. Spark Therapeutics and Pfizer received Breakthrough Therapy Status from the FDA for expedited development of a hemophilia B gene therapy treatment SPK-9001. The treatment is currently also undergoing a Phase 1/2 clinical trial. Link. Alnylam Pharmaceuticals announced positive results of a Phase 1 trial for their subcutaneous therapy fitusiran, designed to treat patients with both hemophilia A and B, with or without inhibitors. Link. Like Share Comment Segment NHF Daily, a digital recap of Annual Meeting. Link WFH The Congress Daily. Link High school swimming star Elijah Warren. Link John Hopkins University article on therapeutic humor. Link BloodStream on Social BloodStream Facebook Page BloodStream Twitter Account BloodStream Host Patrick James Lynch on Twitter and Instagram.