by Madaline Spencer | Jun 24, 2026
Fatima Scipione, Vice President of Global Patient Affairs at Blueprint Medicines, discusses the Colors of SM program. Systemic mastocytosis (SM) is a rare disease usually caused by mutations of the KIT D816V gene. The disorder is characterized by...
by Madaline Spencer | Jun 23, 2026
Eric Lai, MD, Nephrologist at the West Coast Kidney Institute, discusses the importance of early diagnosis in IgA nephropathy (IgAN). IgAN is a kidney disorder that occurs when IgA protein settles in the kidneys. In the early stages, IgAN has no...
by Madaline Spencer | Jun 22, 2026
Andrew Dauber, MD, Chief of Endocrinology at Children’s National Hospital, discusses results from a phase 3 study of Voxzogo (vosoritide) in children with hypochondroplasia. Hypochondroplasia is a rare skeletal disease characterized by very short...
by Madaline Spencer | Jun 19, 2026
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the phase 2 ElevAATe clinical trial of efdoralprin alfa in patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited disease that causes an...
by Madaline Spencer | Jun 18, 2026
Yi Lin, MD, PhD, Hematologist/Oncologist at Mayo Clinic, discusses immune effector cell-associated enterocolitis (IEC-EC) in patients with relapsed/refractory multiple myeloma (R/R MM) treated with ciltacabtagene autoleucel (cilta-cel). MM is a rare...
