February 28 is the 12th Annual Rare Disease Day, the theme of which this year is “bridging health and social care”. This day brings together the rare disease community and highlights the need for better coordination of medical, social, and support services to lessen the burden that rare diseases have …
The Food and Drug Administration’s (FDA’s) Division of Neurology has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019.
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses Acute Hepatic Porphyria (AHPs) and givosiran, an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of AHP.
Mohamad Mohty, MD, discusses the management of hematologic diseases with cellular and immune therapies such as Chimeric antigen receptor (CAR) T cells, which have recently emerged as a novel therapeutic approach, yielding complete responses even in heavily pretreated patients with leukemia, lymphoma, and myeloma.
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s X-linked severe combined immunodeficiency (SCID) program.
The process for researching and developing new medicines is notoriously time-consuming, complicated and costly – and even more so when it comes to therapies for rare diseases.