Spinraza
National Institutes of Health
Papers overview
Semantic Scholar uses AI to extract papers important to this topic.
Aim to report the experience in patients with SMA type 1 treated in the Nusinersen expanded access program (EAP) in a single…
Spinal muscular atrophy (SMA), the most common gentic cause of infantile death caused by mutations in the SMN1 gene, presents a…
Background Classic proximal 5q-SMA is a progressive motor neuron disorder leading to muscle weakness and atrophy and is the most…
KEY POINTS
Canada is one of the few developed countries without a regulatory framework for orphan drugs.[1][1] Orphan drugs are…
SMA is a rare hereditary neuromuscular disease that causes weakness and muscle wasting as a result of the loss of spinal motor…
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease affecting motor neurons, that robs…
Gene therapies featured prominently among this year's newsworthy drugs, some of which have already received a green light from…