The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric scales for adults and the need for modified evaluations to accurately measure ...

If you’ve wondered if spinal muscular atrophy is the same as spina bifida, you aren’t alone. It’s easy to confuse these two conditions since they both involve the spine. But spinal muscular atrophy ...

Spinal muscular atrophy and muscular dystrophy both affect muscle size and function, causing weakness and leading to health complications like difficulty walking, swallowing, and, for some people, ...

In recent years, three disease-modifying therapies have been approved and fully reimbursed in Poland since September 2023. Those therapies have significantly improved the disease outcome but led to ...

Real-world analyses in SMA provide crucial data for long-term comparisons, focusing on SMA types 2 and 3. The HFMSE is a reliable tool for assessing motor abilities and disease progression in SMA2 and ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

For patients with nonambulatory type 2 or type 3 spinal muscular atrophy, apitegromab, a monoclonal antibody that inhibits myostatin activation, improves motor function versus placebo, according to a ...

We all age. And while humanity's life expectancy has increased dramatically in the modern era, we still struggle with the inevitable health issues our bodies face as we get up in the years. For ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic ...

The risk of scoliosis was largely related to what type of SMA a patient had, with those with type III having the lowest risk and those with type II developing scoliosis at a later age than those with ...