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 (SMA). About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Early access to experimental SMA drug apitegromab offered before approval
Disease control NO_LONGER_AVAILABLEThis expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed…
Matched conditions: SMA
Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC