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 ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

In an exclusive interaction with Professor Philip J Young from the University of Warwick we understood the importance of ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

The FDA approved Itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed SMN1 ...

NBS programs are fairly new in the U.S., so not much is known about variations among them or about provider practice patterns for newborns with SMA. In a survey of providers and state NBS programs, ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal ...

The FDA has approved an adeno-associated virus 9 gene replacement therapy to treat individuals aged 2 years and older with ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers ...

Angie Quandt was diagnosed with ALS in late 2022. The disease started in her left foot and has since spread to her legs and voice. Her daughter Emily was finishing college. But instead of moving away, ...