MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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Hope for the youngest: testing SMA drug in Babies' first days
Disease control Recruiting nowThis study aims to understand how the drug risdiplam works in the bodies of very young infants with spinal muscular atrophy (SMA). It will enroll up to 10 newborns under 20 days old to measure the drug's levels in their blood and check for safety. The goal is to gather informatio…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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Race to stop SMA before it starts: new drug trial for babies
Disease control Recruiting nowThis study is testing a drug called salanersen (BIIB115) in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but have not yet shown any symptoms. The goal is to see if starting treatment very early can prevent the onset of SMA symptoms or make them much less s…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated Mar 20, 2026 14:55 UTC
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Second-Chance treatment for babies whose SMA gene therapy stopped working
Disease control Recruiting nowThis study is testing whether the drug risdiplam can help babies and toddlers with spinal muscular atrophy (SMA) who stopped improving or got worse after receiving gene therapy. It will involve 28 children under 2 years old who have two copies of the SMN2 gene. Researchers will m…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE4 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC
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Double treatment hope for SMA babies: gene therapy plus new drug
Disease control Recruiting nowThis study is testing if adding the drug risdiplam to babies who have already received gene therapy for spinal muscular atrophy (SMA) is safe and helps them improve their movement skills. It involves about 28 children under 2 years old. Researchers will measure changes in the bab…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE4 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 17, 2026 13:09 UTC
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First major study launched to track pregnancy outcomes for SMA patients on lifesaving drug
Knowledge-focused Recruiting nowThis study aims to learn about the safety of the SMA drug Spinraza (nusinersen) during pregnancy. Researchers will follow about 20 pregnant women with SMA who received the drug before or during pregnancy, tracking their health and their babies' health for up to two years after bi…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:12 UTC
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Testing an implanted port to deliver SMA medication
Knowledge-focused Recruiting nowThis study aims to understand how the body processes the SMA drug nusinersen when it's delivered through a new implanted port system called ThecaFlex DRx, compared to the standard spinal injection method. The research involves about 58 people with Spinal Muscular Atrophy who are …
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE1 • Sponsor: Biogen • Aim: Knowledge-focused
Last updated Mar 18, 2026 14:41 UTC