by Madaline Spencer | Jul 1, 2024
Márcia Waddington-Cruz, MD, PhD, Federal University of Rio de Janeiro, discusses a Phase 3 trial testing eplontersen in adult patients with hereditary transthyretin polyneuropathy. Hereditary transthyretin amyloidosis (hATTR) is a rare genetic condition...
by Madaline Spencer | Jun 28, 2024
Lisa Berry, Genetic Counselor at Cincinnati Children’s Hospital, discusses results from Fabry patient survey. Fabry disease is a rare lysosomal storage disease. This disease is characterized by a deficiency of the enzyme alpha-galactosidase (alpha-GAL)....
by Madaline Spencer | Jun 27, 2024
The U.S. Food and Drug Administration (FDA) has approved VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). CIDP is a rare neurological disorder...
by Madaline Spencer | Jun 27, 2024
Elisabeth Kugler, PhD, founder of Zeeks, and a patient with a rare disease, discusses her scleroderma and Raynaud’s diagnosis. Scleroderma is a rare connective tissue autoimmune disease that causes inflammation and fibrosis in the skin and other areas of...
by Madaline Spencer | Jun 26, 2024
Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses a series of trials testing the safety and efficacy of investigational drug paltusotine for the treatment of acromegaly. Acromegaly is a rare endocrine disorder resulting from...
