by Madaline Spencer | Jun 11, 2024
Hana Weltin, Intern at Beyond the Diagnosis, discusses how she advocates for herself and others in the rare disease space. Ehlers-Danlos syndromes (EDS) are a group of rare inherited connective tissue disorders. EDS is caused by abnormalities in the...
by Madaline Spencer | Jun 10, 2024
Frank Rivera, Founder of Sarcoidosis of Long Island and Stronger Than Sarcoidosis and patient advocate, discusses how his organization fulfills the unmet needs of sarcoidosis patients. Sarcoidosis is a rare inflammatory disease characterized by the...
by Madaline Spencer | Jun 6, 2024
Dean Suhr, Co-founder and President of the MLD Foundation, highlights how the healthcare system is struggling with the costs associated with expensive therapies, such as the recently approved gene therapy, Lenmeldy (atidarsagene autotemcel), to treat metachromatic...
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,...
