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...
by Peter Ciszewski | Mar 25, 2021
Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary Center and Professor of Medicine at Johns Hopkins discusses treatment options for acromegaly include surgical removal of the adenoma, radiotherapy, or pharmacological reduction of growth hormone...
by Peter Ciszewski | Mar 24, 2021
Tracy McGregor, MD, Senior Director of Clinical Research at Alnylam Pharmaceuticals, discusses medical management options for primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic disease in which excessive oxalate production leads to painful and recurrent...
by Peter Ciszewski | Mar 23, 2021
Sarah Boyce, President and CEO of Avidity Biosciences, gives an overview of facioscapulohumeral muscular dystrophy (FSHD) and the technology her company is developing as a potential treatment for the disease. FSHD is a rare, progressive musculoskeletal disease...
by Peter Ciszewski | Mar 23, 2021
Susan Thornton, CEO of Cutaneous Lymphoma Foundation, discusses how the cutaneous lymphoma community may be impacted by potential COVID-19 vaccines. Cutaneous lymphoma is a rare subtype of non-Hodgkin lymphoma that primarily affects the skin. Cutaneous lymphoma...
