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...
by Madaline Spencer | May 30, 2024
The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb), an interchangeable biosimilar to Soliris (eculizumab). The treatment is approved for both paroxysmal nocturnal hemoglobinuria (PNH), to reduce hemolysis, and atypical hemolytic uremic...
by Madaline Spencer | May 29, 2024
Amanda Cali, Co-founder of Tin Soldiers Global, discusses how the organization helps find patients with fibrodysplasia ossificans progressiva (FOP) and other rare diseases. FOP is a rare disorder in which skeletal muscle and connective tissue are...
by Madaline Spencer | May 27, 2024
Michael Snape, PhD, Chief Science Officer at AMO Pharma, discusses the efficacy of AMO-02 (tideglusib) for myotonic dystrophy type 1 (DM1). DM1 is a genetic disease affecting the muscles and other body systems. Three forms of DM1 may present: the mild...
