Clinical Trials for Muscular Atrophy, Spinal

Find clinical trials for muscular atrophy, spinal. Discover research opportunities and treatment options in your area.

9 Active Trials Found

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A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)

NCT ID: NCT06555419

The PIERRE-PK study is a clinical trial aimed at understanding how the medication nusinersen works in the body when it is delivered using a new device called the ThecaFlex DRx™ System, compared to ...

Recruiting

Phase: PHASE1 Type: interventional Locations: Philadelphia, Pennsylvania, United States, Orange, California, United States +8 more

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT ID: NCT05808764

This clinical trial is studying a medication called risdiplam to see how it works and how safe it is for newborns with spinal muscular atrophy (SMA). SMA is a rare genetic condition that affects mu...

Recruiting

Phase: PHASE2 Type: interventional Locations: Ann Arbor, Michigan, United States, Ottawa, Ontario, Canada +14 more

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

NCT ID: NCT05861986

This clinical trial is studying the effectiveness and safety of a medication called risdiplam in young children with spinal muscular atrophy (SMA) who have already received a gene therapy called on...

Recruiting

Phase: PHASE4 Type: interventional Locations: New York, New York, United States, Philadelphia, Pennsylvania, United States +16 more

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

NCT ID: NCT05861999

This clinical trial is studying the effectiveness and safety of a medication called risdiplam for young children with spinal muscular atrophy (SMA). Specifically, it focuses on children under 2 yea...

Recruiting

Phase: PHASE4 Type: interventional Locations: Philadelphia, Pennsylvania, United States, Dallas, Texas, United States +19 more

A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

NCT ID: NCT05789758

This clinical trial is studying the effects of a medication called Spinraza (nusinersen) in pregnant women who have a condition known as spinal muscular atrophy (SMA). The main goal is to understan...

Recruiting

Type: observational Locations: Bristol, , United Kingdom, Southampton, Hampshire, United Kingdom +8 more

Pediatric Spinal Muscular Atrophy (SMA) China Registry

NCT ID: NCT05042921

The Pediatric Spinal Muscular Atrophy (SMA) China Registry is a clinical trial aimed at understanding how spinal muscular atrophy, specifically the type linked to chromosome 5q (5q-SMA), affects ch...

Recruiting

Type: observational Locations: Guangzhou, Guangdong, China, Changchun, Jilin, China +23 more

Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)

NCT ID: NCT04317794

This clinical trial is studying a medication called Spinraza (nusinersen sodium) to see how safe and effective it is for people with spinal muscular atrophy (SMA) in Korea. The main goal is to moni...

Recruiting

Type: observational Locations: Seoul, , Korea, Republic Of, Daegu, , Korea, Republic Of +23 more

Newborn Screening for Spinal Muscular Atrophy

NCT ID: NCT05481164

This clinical trial is looking into the effectiveness of newborn screening for spinal muscular atrophy (SMA), a serious genetic condition that affects muscle control and movement in infants and you...

Recruiting

Type: observational Locations: Portsmouth, , United Kingdom, Salisbury, , United Kingdom +6 more

Natural History of SMA

NCT ID: NCT05755451

This clinical trial, called the Natural History of SMA, is focused on understanding spinal muscular atrophy (SMA) by observing how the condition affects patients over time. Researchers want to gath...

Recruiting

Type: observational Locations: Rome, , Italy, Rome, , Italy +3 more

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