The mutation that causes Spinal Muscular Atrophy (SMA) can be detected in ... couple who has one child with SMA decides to undergo genetic testing, and both parents are found to be carriers ...

DIAGNOSING: Diagnosing spinal muscular atrophy involves a combination of clinical evaluation, genetic testing, and sometimes additional diagnostic procedures. Given that SMA is a genetic disorder ...

Back to Healio Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease ...

More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...

in treating individuals with spinal muscular atrophy (SMA) Type 2. The randomised, sham-controlled study assessed the efficacy and safety of the gene therapy in treatment-naïve individuals aged ...

The Amsterdam-based biotech firm said that its melt curve analysis-based test is used as a first-tier genetic screening tool to aid the diagnosis of SMA.

Novartis has reported positive phase 3 results with intrathecal dosing of its spinal muscular atrophy (SMA) gene therapy Zolgensma, which could extend the range of children eligible for the drug.

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

Asher Cantrell, diagnosed with SMARD 1, has received experimental gene therapy after social media support and is currently ...

One such condition is Spinal Muscular Atrophy (SMA), a genetic disorder affecting infants and young children, impacting their movement and muscle function. SMA is a genetic disorder of movement ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...