by Madaline Spencer | Jun 6, 2024
Thomas Bartlett, myasthenia gravis patient and Patient Ambassador for the Myasthenia Gravis Foundation of America (MGFA), discusses his personal experience with the disease and keeping hope for future treatment options and care. Myasthenia gravis (MG) is a...
by Madaline Spencer | Jun 5, 2024
Donna Cowan, Associate Director Expanded Access Programs and Registry at Stealth BioTherapeutics, discusses expanded access programs and the company’s drug candidate, elamipretide, for Barth syndrome. Expanded Access Programs Expanded access programs,...
by Madaline Spencer | Jun 4, 2024
David Gusick, Founder of Somebody To Talk To, discusses mental health in patients with rare diseases. Somebody To Talk To focuses on providing mental health resources and support for patients, especially those with Duchenne muscular dystrophy. Through...
by Madaline Spencer | Jun 3, 2024
Cathleen Lutz, PhD, Vice President of the Rare Disease Translational Center at The Jackson Laboratory (JAX), discusses how the laboratory is addressing unmet needs in rare diseases. JAX aims to understand the underlying pathophysiologies of diseases and...
by Madaline Spencer | May 31, 2024
Amy Gray, Chief Executive Officer of the Undiagnosed Diseases Network Foundation (UDNF), discusses the organization and what they do for patients of undiagnosed rare diseases. As Ms. Gray explains, the journey to a diagnosis can be a long and...
