"Monumental" is how Ashley E. Webb, MD Assistant Professor, Department of Pediatrics, Division of Child & Adolescent Neurology Neuromuscular Program Director, UTHealth, University of Texas Health ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

It took some time before Lauren Benz could bring herself to visit a playground with her young son Leonardo. Because of physical limitations resulting from spinal muscular atrophy, watching other ...

The SMA treatment team also includes rehabilitation professionals such as respiratory, physical, occupational, and speech therapists. Rehabilitation treatment involves care related to positioning, ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...

Charlie Zerzan refuses to let SMA define him, using wheelchair basketball to chase championships and inspire others.

This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. 30.4% of patients who received apitegromab had a 3-point ...

The reporting duo demonstrate how drugmakers exploit public funding and legal loopholes to turn high profits, while ...

This is six year old Emma Nowll and like so many kids her age. She is *** prankster. You can also call her. She hates it when I try to help her so um the best thing I can do for her is to just teach ...