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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

00:29:11

An Ally for the Undiagnosed Emerges

RARECast ·

2023/06/29

Amy Gray, CEO of the Undiagnosed Diseases Network Foundation, discusses its work, its relationship with the National Institutes of Health-backed Undiagnosed Disease Network, and the organization’s top priorities.

en

00:23:17

A Patient-Driven Registry Focused on Health-Related...

RARECast ·

2023/06/29

President and CEO of the PKD Foundation Susan Bushnell, Vice President of Research Programs at PKD Foundation Elise Hoover, and Senior Director of Global Strategic Planning for IQVIA’s Integrated Health Practice David Voccola, discuss the new registry, how it is leveraging technology to enable patients to drive insights into their condition, and the challenges it needs to overcome.

en

00:36:22

Help for Rare Disease Patients That’s Just a Click Away

RARECast ·

2023/06/22

Mary Morlino, Global Genes’ RARE Concierge Patient Services manager, discusses her own rare disease journey, the work she’s doing as part of Global Genes’ RARE Concierge program, and the need she is addressing.

en

00:27:31

Diagnosing Autism with a Single Strand of Hair

RARECast ·

2023/06/15

Manish Arora, founder and CEO of Linus Bio, discusses the exposome, how the company’s test for autism works, and how this opens the potential for early intervention. @globalgenes #RARECast

en

00:17:13

Bringing Regenerative Medicine to a Rare Bone...

RARECast ·

2023/06/08

Denis Dufrane, co-founder and CEO of Novadip, discusses the rare pediatric bone disorder congenital pseudarthrosis of the tibia, the company’s cell therapy to address the condition, and how it works.

en

00:26:32

Bringing Precision to CRISPR-Based Genome Editing

RARECast ·

2023/06/01

Rafi Emmanuel, executive vice president of research and development for Emendo Biotherapeutics, discusses the limits of CRISPR today, the company’s experimental program in severe congenital neutropenia, and how the company is engineering CRISPR to optimize it and make it activity precise.

en

00:19:18

Capturing Patients’ Experience in Their Daily Lives

RARECast ·

2023/05/25

Deb Gelinas, neuromuscular executive director at Argenx, discusses myasthenia gravis, the company’s MyRealWorld MG study, and how she hopes the real-world data offers new insights into the rare, autoimmune condition.

en

00:20:03

From One to Many

RARECast ·

2023/05/18

Terry Pirovolakis, who drove the development of a gene therapy for his son’s ultra-rare disease, discusses his newly launched Elpida Therapeutics, its unusual business model, and why it hopes to hand off its therapies at no cost to a partner once they win approval.

en

00:22:08

A Nasal Spray to Curb Excessive Eating in Prader-Willi

RARECast ·

2023/05/11

Seth Lederman, CEO of Tonix, discusses Prader-Willi syndrome, the unmet need for treatments, and why he believes an oxytocin nasal spray has potential to treat the condition.

en

00:26:06

How One Foundation Is Laying the Groundwork to...

RARECast ·

2023/05/04

Pediatrician and mother of a daughter with CACNA1A-related disorder Deborah Ondrasik and CACNA1A Foundation Vice President Sunitha Malepati discuss the ultra-rare neurodegenerative condition, how it progresses, and what the CACNA1A Foundation is doing to advance research to speed the development of treatments and a cure.

en

00:29:20

Forging a New Path for Medicine

RARECast ·

2023/04/27

Amy Dockser Marcus, Pulitzer Prize-winning journalist and author of the new book We the Scientist, discusses how parents of children with the rare lysosomal storage disorder Niemann-Pick disease type C took an active role in the drug development process as citizen scientists, lessons learned from their experience, and how their efforts reflect a broader change in the way biomedical research is conducted.

en

00:41:30

Restoring Hearing through Gene Therapies

RARECast ·

2023/04/20

Laurence Reid, CEO of Decibel, discusses the unmet need in genetic hearing loss, how these conditions can affect early development of children, and the case for the company’s gene therapies to treat these conditions

en

00:34:49

Creating a Sustainable Reimbursement Model for...

RARECast ·

2023/04/13

Rich Horgan, founder and president of Cure Rare Disease, discusses the evolution of his organization, its growing pipeline of therapies, and why developing a viable reimbursement model is essential to creating sustainable development of ultra-rare disease therapies.

en

00:18:02

Lumos Hopes Its Oral Alternative to HGH Will Fuel Its...

RARECast ·

2023/04/05

Rick Hawkins, chairman and CEO of Lumos Pharma, discusses the company’s experimental oral therapy for pediatric growth hormone deficiency, how it works, and why it may be an attractive alternative to existing approaches.

en

00:20:02

Advancing a Gene Therapy for a Rare and Fatal CNS...

RARECast ·

2023/03/30

Samiah Al-Zaidy, vice president of clinical development for Passage Bio, discusses the rare lysosomal storage disorder GM1 gangliosidosis, the company’s experimental therapy to treat the condition, and what’s known about the therapy from work that’s been done to date.

en

00:26:04

Addressing the Barriers to Patient Participation in...

RARECast ·

2023/03/23

Scott Gray, co-founder and CEO of Clincierge, discuses the burdens placed on patients who want to participate in a clinical trial, who Clincierge works to eliminate those, and the impact its work has on recruitment and retention of patients in clinical studies.

en

00:27:23

A Venture Philanthropist Makes the Case for Advocates...

RARECast ·

2023/03/16

Debra Miller, founder and CEO of CureDuchenne, discusses her experience with venture philanthropy, the case for patient advocacy organizations taking equity in exchange for their funding, and what other advocates hoping to stimulate drug development can learn from CureDuchenne’s experience.

en

00:45:13

Seeking a Sustainable Business Developing on N-of-1...

RARECast ·

2023/03/09

The ability to pinpoint the underlying genetic causes of diseases and rapidly generate genetic medicines to address them has created the potential for the development of individualized therapies to treat patients with ultra-rare conditions. EveryOne Medicines is seeking to industrialize this process and scale the development of N-of-1 therapies. We spoke to Irina Antonijevic, chief medical officer of Everyone Medicines, about the company’s business model for pursuing N-of-1 therapies for people with

en

00:15:48

How One Drugmaker Reaches out to Communities at...

RARECast ·

2023/03/03

Pfizer Vice President and North America Medical Lead Younos Abdulsattar and Pfizer Chief Medical Officer of Rare Disease Sonal Bhatia, discuss the rare genetic heart condition ATTR-CM; its disproportionate impact on the Black, African American, and Afro-Caribbean communities; and steps Pfizer is taking to work with community organizations to raise awareness and improve care for people with the condition.

en

00:20:13

A Next-Generation RNA Therapy Targets Telomere Disorders

RARECast ·

2023/03/03

Akihiro Ko, CEO Elixirgen Therapeutics, discusses telomere biology disorders, the company’s self-replicating RNA therapies, and the advantages this new therapeutic approach offers over more traditional mRNA therapies.

en

566 results

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