SMA Newsroom

Background and Objectives of the publication.

Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder associated with continuous motor function loss and complications, such as scoliosis and contractures. Understanding the natural history of SMA is key to demonstrating the long-term outcomes of SMA treatments. This study reviews the natural history of motor function, scoliosis, and contractures in patients with SMA.

We are pleased to announce that, in response to our standing request for important SMA clinical development and programme updates, Roche has shared an update on the RAINBOWFISH clinical trial, for which results from the primary analysis were presented at the 28th World Muscle Society (WMS) Congress.

We are pleased to share with you the latest press release issued by Biohaven. The biopharmaceutical company completes enrolment in Pivotal Phase 3 study of taldefgrobep alfa in spinal muscular atrophy.

We are happy to share with you the latest update shared with us by Scholar Rock.

The company announced yesterday that it has completed enrollment of the SAPPHIRE Phase 3 trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam).

You can read full press release below.