Forest Ray PhD,  —

A wearable cybernetic leg significantly improved the gait and ability to perform walking exercises of people with neuromuscular disorders, such as spinal muscular atrophy (SMA), as compared to a conventional hoist, according to a recent clinical trial. The trial’s results were published in an article, titled “…

Children with spinal muscular atrophy (SMA) who started taking Spinraza (nusinsersen) before symptoms appeared retained their ability to swallow after approximately four years, while youngsters with later-onset SMA walked farther with less fatigue. These results, from analyses performed on multiple ongoing clinical trials, were presented by Biogen,…

Meaningful further improvements in motor skills were evident with apitegromab treatment in later-onset spinal muscular atrophy (SMA) patients on maintenance doses of Spinraza (nusinersen), updated Phase 2 TOPAZ trial findings show. “The TOPAZ results show that apitegromab has promising potential to benefit the large portion of individuals with SMA who still…

Evrysdi (risdiplam) continues to be linked to motor function improvements in a broad range of people with spinal muscular atrophy (SMA), according to recent data from two ongoing Phase 2 clinical trials. These data were presented at the 2021 Virtual SMA Research & Clinical Care Meeting. Presymptomatic…

Evrysdi (risdiplam) has been approved by Health Canada for the at-home treatment of spinal muscular atrophy (SMA) in people ages 2 months and older. “The Health Canada approval of Evrysdi is a welcome addition in our ability to treat SMA. It is an efficacious treatment that significantly increases…

The United States Patent and Trademark Office has granted Scholar Rock a patent for apitegromab, with add-on and combination therapies, for treating muscle conditions such as spinal muscular atrophy (SMA). “We are delighted to have been granted this unique patent that further protects our myostatin approach to…

PTC Therapeutics and the Spinal Muscle Atrophy (SMA) Association will collaborate in funding research into regenerative medicine, and into SMA in particular. With the advent of disease-modifying SMA therapies such as Evrysdi (risdiplam) and Spinraza (nusinersen), regenerative medicine — which aims to restore damage caused by…

Assistive equipment is vital to enhancing function, fun, and social participation in children with severe disabilities such as spinal muscular atrophy (SMA) and should be considered standard-care therapies, along with  medications such as Spinraza (nusinersen). A recent scientific study reviewed the available literature on assistive devices and equipment,…

Patient advocacy groups, a university, and pharmaceutical companies are among the 12 new partners of the European Alliance for Newborn Screening in Spinal Muscular Atrophy, also known as the SMA NBS Alliance. SMA Europe founded the alliance last year in an effort to include SMA in all newborn…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…