MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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New drug shows promise for muscle strength in SMA patients on standard therapy
Disease control CompletedThis study tested a drug called apitegromab in 188 people aged 2 to 21 with later-onset spinal muscular atrophy (SMA types 2 and 3) who were already taking nusinersen or risdiplam. The goal was to see if adding apitegromab could improve muscle function more than a placebo. Partic…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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New hope for SMA kids: extra drug boosts movement after gene therapy
Disease control CompletedThis study looked at whether adding a medicine called nusinersen can help children under 3 with spinal muscular atrophy (SMA) who still have muscle problems even after receiving gene therapy. The 46 children received several doses of nusinersen over about 2 years. Researchers che…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE4 • Sponsor: Biogen • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Massive SMA study reveals Real-World treatment patterns
Knowledge-focused CompletedThis study looked at medical records of over 4,800 people with spinal muscular atrophy (SMA) to see how three approved treatments (onasemnogene abeparvovec, nusinersen, and risdiplam) are used in real life. Researchers tracked complications, medical equipment needs, and hospital …
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated May 17, 2026 04:33 UTC
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Big data reveals Real-World costs and dosing for SMA therapies
Knowledge-focused CompletedThis study analyzed insurance claims from over 4,000 people with spinal muscular atrophy to see how three approved drugs (Zolgensma, Spinraza, and Evrysdi) are actually used in practice and what they cost. Researchers looked at dosing patterns, yearly costs, and overall healthcar…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated May 14, 2026 12:02 UTC
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UK study checks if routine newborn screening can catch rare muscle disease early
Knowledge-focused CompletedThis study looked at whether adding a genetic test for spinal muscular atrophy (SMA) to the standard newborn blood spot screening is practical and accepted by families in the UK. Over 33,000 newborns were screened using leftover blood from their routine Guthrie card. The goal was…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: University of Oxford • Aim: Knowledge-focused
Last updated May 07, 2026 18:39 UTC
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Real-World data reveals how kids with SMA are treated outside clinical trials
Knowledge-focused CompletedThis study analyzed medical records of 213 children with spinal muscular atrophy (SMA) in the Czech and Slovak Republics to see how treatments are actually used in everyday practice. Researchers tracked motor skills, breathing, feeding, and other health measures over time. The go…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated May 04, 2026 16:23 UTC