The FDA granted accelerated approval to Venetoclax (Venclexta) in combination with azacitidine, decitabine or low-dose cytarabine to treat newly diagnosed patients with acute myeloid leukemia (AML) aged 75 years or older.
Daniel de Boer, Founder and CEO of ProQR, discussuses Leber’s congenital amaurosis (LCA), the most common genetic cause of childhood blindness.
The U.S. Food and Drug Administration (FDA) approved Gamifant (emapalumab-lzsg), an interferon gamma (IFNγ) blocking antibody for the treatment of patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.
Roxana Sulica, MD, Associate Professor, Department of Medicine and Director, Pulmonary Hypertension at NYU Langone in New York City discusses the current tretment options of pulmonary arterial hypertension (PAH), a progressive condition that affects the heart and lungs. PAH is characterized by abnormally high blood pressure (hypertension) in the pulmonary …
Rachelle Dixon, President and co-founder’s of the HSAN1E Society discusses hereditary sensory and autonomic neuropathy type 1E (HSAN1E), a progressive disorder of the central and peripheral nervous systems.