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PODCAST

RARECast

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.

business

All Episodes

00:27:39

In Pursuit of a Treatment for His Son, A Lawyer...

RARECast ·

2023/11/22

Alex Nemiroff, general counsel at Praxis Precision Medicines, discusses his journey as a parent of a child with a rare, genetic disease; how he came to launch a biotechnology company to develop a treatment for his son, and what other patient families looking to advance a therapy can learn from his experience.

en

00:32:49

In Push for Treatment, a Patient Organization Becomes...

RARECast ·

2023/11/16

Marianne Clancy, executive director and senior director of strategic partnerships for Cure HHT, discusses the organization’s decision to sponsor a clinical trial, why it felt it was necessary to do so, and what other patient organizations can learn from its experience.

en

00:31:54

Delayed Diagnosis of Rare Diseases Takes Staggering...

RARECast ·

2023/11/09

Annie Kennedy, chief of policy, advocacy, and patient engagement for the Everylife Foundation; discusses the diagnostic odyssey, the economic toll it takes, and what can be done to shorten the time to a diagnosis.

en

00:22:58

A Cell Therapy to Control Heart-Damaging Inflammation...

RARECast ·

2023/11/02

Linda Marban, CEO of Capricor Therapeutics, about Duchenne muscular dystrophy, the damage the condition does to the heart, and how the company’s experimental cell therapy modulates the immune system to control chronic inflammation caused by the disease.

en

00:21:50

Stimulating the Growth of Muscle Mass in People with...

RARECast ·

2023/10/26

Lindsey Lee Lair, Biohaven’s vice president of clinical development, discusses spinal muscular atrophy, the progress we’ve seen in treating the condition, and the company’s efforts to develop a therapy to stimulate the growth of muscle mass and strength in people with the disease.

en

00:45:18

Turning Grief into Action

RARECast ·

2023/10/19

Ron Garber, co-founder and board president of the Yaya Foundation, discusses 4H leukodystrophy, how the organization built a research agenda, and the rapid progress it has made.

en

00:22:56

A Plea to the FDA to Give an Ultra-Rare Disease Drug...

RARECast ·

2023/10/12

Shelley Bowen, co-founder and director of family services for the Barth Syndrome Foundation, about Barth syndrome, the fight over approval for the first drug to treat the condition, and why it points to a systemic problem with the lack of consistent use of the flexibility Congress granted the FDA to get treatments to patients with ultra-rare diseases.

en

00:44:35

How the Woolly Mammoth Beat a Path to Better Gene...

RARECast ·

2023/10/05

Claire Aldridge, chief strategy officer for Form Bio, discusses the challenges of developing gene therapies, how Form’s AI platform can address that, and how it works grows out of tools that were developed to bring the woolly mammoth back from extinction.

en

00:26:12

A Genetic Counselor’s Journey into Patient Advocacy

RARECast ·

2023/09/28

Genetic counselor and mother of a child with the rare, genetic disease neurofibromatosis type 1 Danielle Bonadies discusses caring for a child with the condition, how her professional and private lives have been thrust together because of her son’s diagnosis, and her evolution as a patient advocate.

en

00:30:29

A Company in the Fold of Rare Disease

RARECast ·

2023/09/21

Matthias Alder, CEO of Gain Therapeutics, discusses the role protein misfolding plays in a range of diseases, Gain’s platform technology, and its lead experimental therapy in development to treat Gaucher disease.

en

00:27:39

Disabling Antibodies that Drive Rare, Immunological...

RARECast ·

2023/09/14

Matthew Shaulis, chief commercial officer and U.S. president for Hansa Biopharma, discusses the company’s antibody-cleaving platform technology, how it works, and its range of potential uses.

en

00:19:26

Keeping the Sentinels of the Brain Vigilant

RARECast ·

2023/09/07

Ivana Magovčević-Liebisch, president and CEO of Vigil Neuroscience, discusses the role of microglia, what happens to these immune cells in the rare, neurodegenerative condition ALSP; and why the company’s therapeutic approach can have implications for a range of rare and common neurodegenerative conditions.

en

00:23:44

Considering the Unfinished Work in Cystic Fibrosis

RARECast ·

2023/08/31

Jamie Chang, senior medical director at the contract research organization Rho, discusses the advances that have been made to treat cystic fibrosis, the treatment gap that remains, and what’s working its way through the pipeline that may change that

en

00:28:55

How to Advocate for Yourself as You Battle Cancer

RARECast ·

2023/08/24

Bill Potts, five-time cancer survivor and author of Up for the Fight, discusses why it’s essential for cancer patients to learn to advocate for themselves, how to navigate the mental and emotional aspects of the disease, and why it’s important to stay motivated and celebrate small achievements along the way.

en

00:25:34

Targeting Neuroinflammation to Treat ALS

RARECast ·

2023/08/17

Howard Berman, chairman and CEO of Coya Therapeutics, discusses the role of inflammation in neurodegenerative conditions, regulatory T cells, and the company’s experimental therapy to treat ALS.

en

00:33:42

Leveraging Community Partnerships to Address a Rare...

RARECast ·

2023/08/10

Connie Lee, president and CEO of the Alliance to Cure Cavernous Malformation, discusses the Baca Family Historical Project, how her organization leveraged partnerships with community leaders to engage the community to identify people with a potentially fatal rare condition, and how the organization used a novel approach to improve their access to testing and care.

en

00:32:18

Accelerating Gene Editing Therapies for Rare,...

RARECast ·

2023/08/03

Steve Murray, associate professor at The Jackson Laboratory, discusses the promise of gene-editing therapies, the work the lab is leading grant under a $22.8 million NIH grant to develop four gene-editing therapies for rare neurological conditions, and why the work could have broad implications for similar conditions.

en

00:19:15

Inhibiting Electrical Activity in Rare, Seizure...

RARECast ·

2023/07/27

Alex Aimetti, chief scientific officer of Marinus Pharmaceuticals, discusses the company’s drug Ztalmy to treat seizures in people with CDKL5 deficiency disorder, how it works, and the efforts to expand its use to other seizure disorders.

en

00:23:49

Improving the Function and Usability of Clothing for...

RARECast ·

2023/07/20

Yasmin Keats, executive director of Open Style Lab, about her organization’s efforts to promote the design of adaptive fashion, the impact it can have on work and other opportunities for people living with disabilities, and how it is helping to change the fashion industry.

en

00:24:17

Treating Cardiopulmonary Disease with Inhaled...

RARECast ·

2023/07/13

Josh Ziel, chief operating officer and interim CEO of Aerami, discusses pulmonary arterial hypertension, the company’s experimental therapy to treat the condition, and the company’s precision inhalation technology, and its efforts to build a pipeline of therapies that make use of its proprietary inhalation technology.

en

566 results

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