Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

Leptin merely correlated with body mass index, the study found. A new report on patients with types II and III spinal muscular atrophy (SMA) has found that levels of the peptide hormone leptin are ...

Qualitative interviews conducted with caregivers of children with spinal muscular atrophy (SMA) found that the symptoms with the greatest effect on disease burden were not always the most prevalent ...

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 ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

MIAMI - Celine Domalski was busy riding her unicorn at her Miami Beach condo as she readied to walk in a very important fashion show. "I'm doing a fashion show," Celine told Lisa Petrillo. "Are you ...

"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 ...

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

Spinal muscular atrophy (SMA) is a group of serious, progressive diseases that destroys motor neuron cells. SMA treatment aims to reduce symptoms and slow or stop the progression of the disease. SMA ...

SMA type 3 is a hereditary disease that affects motor neurons. People may also refer to it as Kugelberg-Welander disease. Symptoms usually begin after 18 months of age. Children with SMA type 3 can ...