Pre-symptomatic salanersen study for babies with SMA
Treatments studied
Part of Brain & nervous system clinical trials.
This trial tests if the drug salanersen can help movement and is safe when given to babies diagnosed with spinal muscular atrophy before they show symptoms. It's for very young infants with specific genetic types of SMA.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your baby must be 42 days old or younger when starting the drug.
- Genetic testing must show the type of SMA caused by a missing or changed gene on chromosome 5.
- Your baby must have 2 or 3 copies of the SMN2 gene.
- Nerve signals in the arm must be at a certain level on testing.
- Your baby's weight must be at or above the 3rd percentile for their age.
- Your baby must not have any symptoms of SMA yet, like muscle weakness or breathing problems.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study tests a new medicine called salanersen in babies who have already received gene therapy for SMA. It aims to see if adding salanersen is safe and effective.
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
This study looks at how Spinraza (nusinersen) taken during or before pregnancy affects people with 5q spinal muscular atrophy (SMA) and the health of their babies. It mainly fits those who were pregnant and received Spinraza around the time of conception or during pregnancy.
This trial tests a medicine called salanersen (BIIB115) to see how well it improves movement and how safe it is in people with spinal muscular atrophy (SMA). It includes those who have never taken an SMA drug or are currently on risdiplam and willing to switch.
This Phase 1/2 study tests whether EXG001-307 is safe and may improve health in babies with spinal muscular atrophy (SMA) type 1. It mainly looks at safety first, and whether the treatment can help the body work better over time.
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