RARECast

RARECast
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RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

  1. -4 ДН.

    A Call on the FDA for Timely Reviews of Life-Saving Therapies for Rare Diseases

    Hunter syndrome is caused by the body’s inability to produce a critical enzyme needed to break down cellular waste. The condition can cause damage to organs throughout the body as well as to the brain. A new generation of therapies in development, including a gene therapy currently under review by the U.S. Food and Drug Administration, that has the potential to address the neurological symptoms of the disease. Still, patient advocates have been frustrated by regulatory delays and are seeking to push the FDA and Congress to take action. We spoke with Kristin McKay, CEO of the Hunter syndrome patient advocacy organization Project Alive, about the need for new therapies, the importance of early detection, and the patient community’s concerns with regulatory delays in approving needed treatments. An editor’s note: Since recording this podcast, the FDA granted accelerated approval to Stealth Biotherapeutics’ Forzinity for Barth syndrome, which is referenced in the discussion.

    27 мин.

  2. 2 ОКТ.

    The Need to Look Beyond Seizures When Treating DEEs

    Developmental and epileptic encephalopathies are a group of rare disorders that are characterized by frequent seizures that often don’t respond to existing medications. These are complex conditions that involve progressive cognitive and behavioral manifestations that can pose significant burdens on patients and their families. In both clinical practice and within the biopharmaceutical industry, there has been a tendency to focus on seizure control, while often overlooking the non-seizure burdens of developmental and epileptic encephalopathies. We spoke to Amelie Lothe, global medical community head for rare epilepsies at UCB, about the need to view these developmental and epileptic encephalopathies as complex neurodevelopmental conditions, the need to go beyond seizure frequency when it comes to clinical trial outcome measures, and what drug developers can do to improve their research focus to include broader patient and caregiver experiences.

    32 мин.

  3. 25 СЕНТ.

    Taking On Big Competitors with an Oral Therapy to Treat Achondroplasia

    Achondroplasia is the most common form of dwarfism. Beyond short stature, people living with achondroplasia can experience serious health complications, including compression of the brainstem and upper spinal cord due to impaired development of the skull. Tyra Biosciences is developing a next-generation medicine to precisely target FGFR3, an overactive growth factor that causes achondroplasia. We spoke to Todd Harris, CEO of Tyra Biosciences, about the company’s experimental once-daily, oral medicine for achondroplasia; what’s known about it from studies conducted to date, and why he believes it will offer competitive advantages over existing therapies.

    23 мин.

  4. 18 СЕНТ.

    Going Viral to Fight Brain Cancer

    Recurrent high-grade glioblastoma is a rare and aggressive brain tumor, which today is generally treated with surgery and chemotherapy. Outcomes are poor, with survival ranging from three to nine months and five-year survival rates less than 10 percent. Candel Therapeutics is developing viral immunotherapies that both kill tumor cells directly and enlist the patient’s own immune system in the fight against cancer. It’s experimental therapy CAN-3110 uses a modified herpes simplex virus that carries a viral gene that is designed to allow the virus to replicate in tumor cells while avoiding healthy cells. We spoke to Paul Peter Tak, president and CEO of Candel, about its viral immunotherapy, how it works, and what clinical studies have shown to date.

    28 мин.

  5. 11 СЕНТ.

    Changing the Playbook on Childhood Cancers

    While there has been enormous innovation in the treatment of cancer over the past two decades, much of this has been focused on adult cancers. Despite the advent of targeted therapies and immunotherapies, the treatment of childhood cancers relies largely on chemotherapy and radiation, both of which can create lifelong side effects in developing bodies. And cancer remains the leading cause of death by disease in children in the United States and the United Kingdom. C-Further, an international consortium created by LifeArc and Cancer Research Horizons, is working to advance innovative treatments for childhood cancers. It not only provides funding to discover and develop transformative therapies to treat childhood cancers but also leverages its network to help advance promising therapies. We spoke to David Jenkinson, head of childhood cancer translational challenge at LifeArc, about the approach C-Further is taking, the scientific and economic challenges of developing treatments for childhood cancers, and why new models for advancing these therapies are needed.

    29 мин.

  6. 4 СЕНТ.

    Sometimes the Hoofbeats Do Belong to Zebras

    Avion was a healthy and athletic 15-year-old who became critically ill when he was admitted into a pediatric intensive care unit. For Robin Williams, assistant professor of pediatric hematology/oncology at the University of Minnesota Masonic Children's Hospital, Avion offered a medical puzzle she couldn’t crack on her own. His immune system was on overdrive and it was attacking healthy cells and organs within his body. Though testing ruled out blood cancers, it was only when Williams consulted a friend outside the hospital that she realized Avion was suffering from TAFRO, a subtype of the ultra-rare disorder idiopathic multicentric Castleman’s disease, a condition that has characteristics of both blood cancers and autoimmune disease. We spoke to Williams about the challenges physicians face in diagnosing patients with rare diseases, the thought process she went through in Avion’s case, and why she’s working to educate other physicians about the ultra-rare condition.

    33 мин.

  7. 28 АВГ.

    Breathing Easier with a Breakthrough Immunotherapy

    Recurrent respiratory papillomatosis is a potentially life-threatening disease of the upper and lower respiratory tract caused by chronic infection with human papillomavirus type 6 or type 11. In the absence of approved therapies, people with the condition often undergo repeated surgeries to clear their airways. The U.S. Food and Drug Administration in August approved Precigen’s Papzimeos, an immunotherapy that targets the underlying cause of the RRP, as the first approved therapy to treat the condition. We spoke to Kim McClellan, president of the Recurrent Respiratory Papillomatosis Foundation and Simon Best, associate professor of otolaryngology-head and new surgery at Johns Hopkins Medicine, about recurrent respiratory papillomatosis, the daily impact the condition can have on the lives of people with the disease, and what the approval of this therapy means for people living with the condition.

    30 мин.

  8. 21 АВГ.

    Using AI Open Innovation to Tackle ALS

    Tris Dyson founded Challenge Works to incentivize innovators to solve societal problems. Dyson, who was diagnosed with amyotrophic lateral sclerosis, is now using the platform to find new treatments for the progressive neurodegenerative disease. The $10 million Challenge Works' Longitude Prize on ALS harnesses AI, open collaboration, and big data to find new treatments for the condition. We spoke to Dyson, managing director of Challenge Works, about his diagnosis of ALS, the case for using a prize to spur innovation, and the potential for leveraging AI to find treatments for the disease.

    28 мин.
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RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

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  • Автор
    RARECast
  • Годы выхода
    2014 - 2025
  • Выпуски
    564
  • Ограничения
    Без ненормативной лексики
  • Авторские права
    © All rights reserved
  • Сайт подкаста
    RARECast

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