MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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New drug could help babies with SMA walk and sit normally
Disease control Recruiting nowThis study tests a drug called salanersen in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug helps the body make more of a protein that is missing in SMA, which is needed for muscles to work. Researchers want to see if starting t…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Can a second treatment help kids with SMA who stalled after gene therapy?
Disease control Recruiting nowThis study tests whether adding risdiplam, a daily oral medicine, can help children under 2 with spinal muscular atrophy (SMA) who stopped getting better or started declining after receiving gene therapy. The study will enroll 28 children and measure changes in motor skills over …
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE4 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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New drug may boost motor skills in kids with SMA after gene therapy
Disease control Recruiting nowThis study is testing whether the drug risdiplam can improve motor skills in children under 2 with spinal muscular atrophy (SMA) who have already received gene therapy. The 28 participants will take risdiplam by mouth, and researchers will measure changes in their gross motor ski…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE4 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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New implant could change how SMA patients get their medicine
Disease control Recruiting nowThis study looks at how the body handles the drug nusinersen when given through a new implantable device called ThecaFlex DRx™, compared to the usual method of a lumbar puncture (spinal tap). About 58 people with spinal muscular atrophy (SMA) who are already in another study (PIE…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE1 • Sponsor: Biogen • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New study tracks spinraza in pregnancy for SMA moms
Knowledge-focused Recruiting nowThis study gathers health information from pregnant women with spinal muscular atrophy (SMA) who have taken the drug Spinraza (nusinersen), and from their babies. Researchers want to learn about pregnancy outcomes, such as miscarriage, birth defects, and infant development. The s…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC