SMA
Clinical trials for SMA explained in plain language.
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New drug shows promise for SMA patients in major trial
Disease control OngoingThis study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy. About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The goal is to see if the…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 18, 2026 12:02 UTC
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New hope for SMA patients: early access to apitegromab
Disease control NO_LONGER_AVAILABLEThis program gives eligible patients with spinal muscular atrophy (SMA) early access to the investigational drug apitegromab before it is officially approved. Participants must be at least 2 years old and have a confirmed SMA diagnosis. A doctor will decide if the potential benef…
Matched conditions: SMA
Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated May 15, 2026 11:53 UTC