RARECast

Noreen Henig, chief medical officer for Kezar Life Sciences, discusses lupus nephritis, the company’s first-in-class candidate to target the condition, and why it may have application in a range of autoimmune conditions.

Ben Munoz, co-founder of Ben’s Friends, discusses his own experience with a rare condition, the role support from someone with the same condition has played for him, and his efforts to create a forum where patients with specific conditions can connect and provide support to each other.

Timothy Yu, a neurologist and attending physician in the Division of Genetics and Genomics at Boston Children’s Hospital Yu, discusses his work to advance individualized therapies for rare disease patients, the potential to industrialize the process, and the challenges that need to be addressed.

Clinton Moore, Cystinosis Research Network and co-executive producer of the film Walk in My Shoes, on his son’s rare disease journey, his own emergence as an advocate, and how he came to recognize the power an individual has to make a difference.

Thom Rossi, CEO of Venthera, discusses the company’s lead experimental therapy, its first-in-man-trial, and how it is leveraging the resources of BridgeBio.

Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation, discusses how her organization is preparing the patient community for the availability of the first therapies for the condition, the issues people will need to consider, and how families may need to think differently about their futures.

Cara O’Neill, chief scientific officer of Cure Sanfilippo Foundation, discusses Sanfilippo syndrome, her journey from physician to advocate, and her efforts to craft a research agenda.

Raj Kannan, CEO of Chiasma, discusses the company’s recently approved therapy for acromegaly, the company’s drug delivery technology, and the value of having oral alternatives to injectable therapies.

Colleen Huysman, a clinical social worker with the Bridges Adult Transition Program at Boston Children’s Hospital, and Sneha Dave, founder and executive director of the Health Advocacy Summit, discuss the challenges young adults with rare and chronic condition face as they seek their own health insurance for the first time.