by Peter Ciszewski | Mar 30, 2021
Susan Thornton, CEO of Cutaneous Lymphoma Foundation, gives an overview of the foundation. Cutaneous lymphoma is a rare subtype of non-Hodgkin lymphoma that primarily affects the skin. Cutaneous lymphoma can originate in B-cells (i.e., cutaneous B-cell...
by Peter Ciszewski | Mar 30, 2021
Tracy McGregor, MD, Senior Director of Clinical Research at Alnylam Pharmaceuticals, gives an overview of primary hyperoxaluria type 1 (PH1). As Dr. McGregor explains, PH1 is a rare genetic disease in which excessive oxalate production leads to painful and...
by Peter Ciszewski | Mar 30, 2021
Amy Bekier, facioscapulohumeral muscular dystrophy (FSHD) patient and Board Member of the FSHD Society, talks about how she manages FSHD. FSHD is a rare, progressive musculoskeletal disease in which persons initially lose muscle strength in the face, shoulders,...
by Peter Ciszewski | Mar 26, 2021
Chris Peetz, President and CEO of Mirum Pharmaceuticals, discusses the investigational drug, maralixibat that is being developed to treat alagille syndrome. Alagille syndrome is an inherited disorder that can affect the liver, heart, skeleton, eyes, and...
by Peter Ciszewski | Mar 25, 2021
Malika Abrams, a young woman with sickle cell anemia and avascular necrosis (AVN), talks about her work in rare disease advocacy. Sickle cell anemia is an inherited blood disorder. Early symptoms usually occur in childhood and include swelling, fatigue, and...
