Podcasts about rdeb

Welcome to HCPLive's 5 Stories in Under 5—your quick, must-know recap of the top 5 healthcare stories from the past week, all in under 5 minutes. Stay informed, stay ahead, and let's dive into the latest updates impacting clinicians and healthcare providers like you! Interested in a more traditional, text rundown? Check out the HCPFive! Top 5 Healthcare Headlines for April 21-April 27, 2025: FDA Approves Pz-cel (Zevaskyn) Gene Therapy for RDEB The FDA has approved pz-cel, the first autologous gene therapy for RDEB, following pivotal Phase 3 data demonstrating efficacy and safety in wound healing. FDA Approves Upadacitinib, Expanding Treatment for Adults With Giant Cell Arteritis Upadacitinib has been approved for giant cell arteritis, supported by Phase 3 data showing its potential to induce sustained remission and reduce corticosteroid reliance. FDA Approves Nipocalimab Generalized Myasthenia Gravis for Adults, Children Nipocalimab received FDA approval for gMG in antibody-positive patients aged ≥12, expanding therapeutic options across major serotypes. Semaglutide Improves Steatohepatitis, Fibrosis in Phase 3 MASH Trial Phase 3 trial results show semaglutide significantly improves steatohepatitis and fibrosis markers in patients with MASH, without worsening liver histology. Increased Fasting Blood Glucose Triples Risk of Heart Damage in Adolescents International data links elevated fasting glucose and insulin resistance in adolescence to markedly increased future risk of heart damage, especially among females.

Novelette is 38 year old woman, living with a life limiting illness, called Epidermolysis Bullosa. I have the Recessive Dystrophic form of Epidermolysis Bullosa, RDEB. I live in Canada. I have a specialist in philosophy from the University of Toronto. Since 2018, I have been a trained hospice and peer bereavement Volunteer. I co-facilitate a lot of peer bereavement groups. I do a lot of mindfulness for grieving loss groups. I also help with training new hospice and peer bereavement volunteers. I've also volunteered as a hospice care companion to others, living with a life limiting illness.  I enjoy writing poetry and painting. Talking walks in nature. And I enjoy spending time with my nieces and nephew and caring for my cat.

We are rounding out EB Awareness Week with this Monday's Podcast Rerelease. Originally airing in April of 2021, this Abeona Sponsored Webinar, Gene Therapy for Large Chronic Wounds in Recessive Dystrophic Epidermolysis Bullosa, was presented by Jean Tang, MD, PhD. Dr. Tang provided an update on the EB-101 pivotal Phase 3 VIITAL study and answered questions from the audience.To view the original webinar, please click here.

Recessive dystrophic epidermolysis bullosa is a rare, genetic, progressive condition caused by the deficiency of collagen type VII. People with severe cases of the condition suffer from blistering, vision loss, disfigurement, and other serious medical problems. Castle Creek Biosciences is developing a therapy that involves genetically modifying a patient's own fibroblasts—the cells in the connective tissue—to get them to produce collagen VII. The modified cells are injected where needed and can be dosed repeatedly. We spoke to Matthew Gantz, president and CEO of Castle Creek, about the company's experimental therapy for RDEB, how it works, and how the company is building out its pipeline through dealmaking.

Brady is a five-year-old who is living with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin condition. His mother, Eileen, describes what it was like to discover that Brady has RDEB and tells us how she is both the mother of a brave, happy little boy and the nurse who causes him pain during his daily wound care. RDEB has changed their lives. Sanuj Ravindran, M.D., executive chairman of BridgeBio's Phoenix Tissue Repair, joins the conversation to talk about the condition and explain how EB affects the body internally and externally.

In this episode, Annie, Certified Child Life Specialist, talks to Eilieen. She is the amazing mom of a determined, fun loving, 4-year-old hero named Brady. Brady was diagnosed with Recessive Dystrophic Epidermolysis Bullosa (RDEB). While he inspires all who meets him, his favorite thing is just being a typical boy and being treated as such. After a move from Texas to Colorado to find the best care and environment for him, life shifted and their family became advocates in the RDEB community. Eileen is on the board of the EB Research Partnership and they have an absolutely star studded (like for real a ton of A-listers) event coming up on November 18th that will be streamed on Amazon.  EB Research - On November 18th, 2020 at 8pm EST, we will be hosting an extraordinary fundraiser with several A-List celebrities, and streamed on Amazon!!! This is an unparalleled  opportunity for others to learn about Brady's condition, and get us closer and closer to our goal for a cure. Learn more here!!   DEBRA is the organization talked about that welcomes donations of supplies etc for children with EB Follow along with Eileen and Brody on Instagram and Facebook. Find more resources, parent stories and support on www.childlifepodcast.com. Child Life On Call | Instagram | Facebook | Twitter

On Episode 12 (the final episode in Series 1) we have the patient ambassador for DEBRA Ireland, Emma Fogarty. Emma suffers from RDEB, also known as Recessive Dystrophic Epidermolysis Bullosa. EB is a rare, genetic skin condition which causes the skin to blister at the slightest touch. These wounds, some of which will never heal, occur both internally and externally and are constantly at risk of severe infection. Children that are born with EB are highly susceptible to a very aggressive form of skin cancer from as early as their teenage years and there is currently no cure for EB. Emma has an extraordinary life, and has experienced pain most humans can't even fathom. This remarkable woman is everything The Other Side Of Perfect is about because, she's not defined by her struggle, instead her strength. In this episode we discuss in detail, what it's like living with EB, life outside the condition, the incredible people Emma has met on her journey, and why fundraising is so important. On the topic of fundraising, DEBRA Ireland is the only charity devoted solely to the care and cure of EB. DEBRA supports patients and their families living with the devastating effects of this condition and helps them cope with all the challenges that it brings. DEBRA provides this through their in-home EB Community Care Programme as well as advancing research both nationally and internationally. In February 2020 I will be taking part in the DEBRA Ireland Arctic Challenge. My mission is to survive in the harsh environment of Northern Finland, 150km above the Arctic Circle, totally off grid with no running water or electricity. The aim is to learn new skills and survive the extreme cold of the arctic regions whilst raising money for children living with EB. Last year the trip raised just under €250,000 from just 35 participants. In 2020 there's 36 of us heading and it's the first year men will be joining the mission. I know you will take a huge amount from this episode and I urge you to use the emotion and empathy experienced to make just a small donation where 100% of the proceeds from my Arctic fundraising go directly to DEBRA Ireland.DONATIONS https://www.justgiving.com/fundraising/joannelarbyArctic Challenge https://www.youtube.com/watch?v=i0AnSVhxHVEDEBRA Ireland https://debraireland.org/Visit https://theothersideofperfect.com/

11 March 2014: In this Skinpod episode Dr. John McGrath, from King's College London, discusses fibroblast cell therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB).

Jeanine Cook-Gerard from the Department of Nursing talks with John Maslowski, President and Chief Executive Officer of Fibrocell, about the rare genetic skin disease Recessive Dystrophic Epidermolysis Bullosa (RDEB). John describes Fibrocell’s main objective - to find a cure for RDEB and announces that the FDA is now allowing the second phase of this clinical trial in which Fibrocell will be enrolling six pediatric patients ages 7 and older.

Jeanine Cook-Gerard from the Department of Nursing talks with John Maslowski, President and Chief Executive Officer of Fibrocell, about the rare genetic skin disease Recessive Dystrophic Epidermolysis Bullosa (RDEB). John describes Fibrocell’s main objective - to find a cure for RDEB and announces that the FDA is now allowing the second phase of this clinical trial in which Fibrocell will be enrolling six pediatric patients ages 7 and older.

Epidermolysis Bullosa is a rare genetic skin disorder. One thing to describe its rareness is that a doctor or a nurse can be working a lifetime and never bump into this condition. Because of this, there is a lot unknown about the condition and most Doctors are unfamiliar with the condition. I will try to answer some of the most common questions regarding the condition and perhaps clear up some misconceptions. It is estimated that about 10,000 Americans, mostly kids, have some form of EB. With modern medical care, some with the worse kinds of EB can live into their thirties. Unfortunately, by this age most will succumb to a particularly aggressive skin cancer (Squamous Cell Carcinoma) that is somehow touched off by EB. Of these 10,000 effected, less than 300 have the same kind as my son Nicky (he has the Hallopeau-Siemens subtype, also called Severe Generalized), making the RDEB form so rare than only one out of every one million babies is born with it.

About Jennifer in her own words: My name is Jennifer (Lyric) Bolles. I was born in FL and have lived in many different states throughout my life. Currently, and indefinitely, I am in Cincinnati, OH. I moved here almost 6 years ago for the EB drs at Cincinnati Children's Hospital and Medical Center's (CCHMC) EB center. My health was drastically suffering as a result of the arrogant, ignorant FL drs who refused to learn about EB I am 42 yrs old and have Recessive Dystrophic Epidermolysis Bullosa. My subtype of RDEB is Inversa, an extremely rare subtype of RDEB. I am externally affected by this subtype of RDEB but my chronic areas are mostly where skin rubs/meets skin. I am more affected internally (oral, esophageal, GI etc...). As a child/young adult, I was active in choral groups, ballet, gymnastics and track but not without injuries. I kept trying and pushing myself until my 2nd year of track in high school. Ultimately, I did quit track as a result of falls that caused severe damage all over my body which took weeks to heal each time. It's one of the rare times in my life that I was truly emotionally devastated by having EB. I began working after high school in retail, food service, clerical positions and found my way to my true love, working with children, specifically special need children, typically ADD/ADHD, mental, emotional, behavioral issues, victims of abuse and neurological issues. I've been working in Child Development for almost 20 years. I am only able to work part time as a result of EB and Fibromyalgia so I typically work with private families who need PT or occasional care.

Fibrocell Science is developing cell and gene therapy to treat rare skin and connective tissue diseases. The company recently reported encouraging pre-clinical results on its gene therapy for RDEB a congenital, progressive, and debilitating genetic disorder that leads to death. The data sets the stage for Fibrocell and its partner Intrexon to advance the experimental therapy to human clinical trials. We spoke to David Pernock, chairman and CEO of Fibrocell about the company, its cell and gene therapies, and how these technologies promise to bring new approaches to treating devastating diseases.