by Madaline Spencer | Jan 21, 2025
Marina Kremyanskaya, MD, PhD, Icahn School of Medicine at Mount Sinai, discusses the development of gene silencer divesiran for treatment of polycythemia vera (PV). PV is a myeloproliferative disorder characterized by an elevated absolute red blood cell...
by Madaline Spencer | Jan 20, 2025
Keyur B. Shah, MD, Virginia Commonwealth University Health, discusses the approval of acoramidis for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a rare amyloidosis caused by aging or genetic mutations. This...
by Madaline Spencer | Jan 17, 2025
Nora Sandorfi, MD, Professor of Clinical Medicine, Rheumatology at the University of Pennsylvania, discusses patient sentiments regarding treatment options for warm autoimmune hemolytic anemia (wAIHA). wAIHA is a rare, autoimmune disorder in which...
by Madaline Spencer | Jan 16, 2025
Michael Parini, CEO of Spur Therapeutics, discusses SPR301, an investigational gene therapy being developed for patients with Parkinson’s disease with GBA1 gene mutations. Parkinson’s disease is a neurological disorder causing unintended or...
by Madaline Spencer | Jan 15, 2025
Andrés Klein, PhD, Associate Professor and Director of the Doctorate Program in Sciences and Innovation in Medicine at Universidad del Desarrollo, Santiago, Chile, discusses the identification of genetic modifiers for treatment in lysosomal disorders. ...
