Researchers in Japan are conducting a study to better understand the natural history of spinal muscular atrophy (SMA) and how well disease-modifying therapies work in the real-world setting. The Biogen Japan-sponsored study will draw on data from the Japan Registry for Adult Subjects of Spinal Muscular Atrophy (jREACT-SMA), which…
Leah Zelaya, a 15-year-old with a rare form of spinal muscular atrophy (SMA), was chosen by the Muscular Dystrophy Association (MDA) to be its 2023 MDA National Ambassador. As national ambassador, the Brooklyn, New York, teenager represents all those living with a neurological disease in the U.S. She and…
Spinraza (nusinersen) treatment is safe in children with spinal muscular atrophy (SMA) and does not cause unwanted immune responses, a study confirmed. The study, involving case reports on three children with SMA who developed elevated levels of white blood cells after Spinraza treatment, also found no changes in levels…
Newborn screening for spinal muscular atrophy (SMA), coupled with early access to disease-modifying therapies, can improve the likelihood that children with the disease will be able to walk, a new study suggests. Also, fewer patients in the newborn screening group tended to require ventilatory or feeding support,…
Treatment with the muscle strengthener pyridostigmine may reduce patient-reported fatigue among people with spinal muscular atrophy (SMA), results from a small clinical trial suggest. Still, the treatment failed to ease motor fatigue and improve motor function compared with a placebo — which were the main goals of the…
In an agreement meant to provide Ionis Pharmaceuticals with additional money to advance potential therapies for various diseases, the company is giving Royalty Pharma a percentage of the payments it receives from sales of Spinraza (nusinersen), an approved therapy for spinal muscular atrophy (SMA). In exchange, …
The ongoing SAPPHIRE clinical trial testing apitegromab, Scholar Rock’s investigational muscle-directed therapy, in people with spinal muscular atrophy (SMA) is expected to complete patient enrollment later this year, the company announced. Top-line data from the trial are anticipated in 2024. Should results be positive, Scholar Rock expects…
CANbridge Pharmaceuticals has acquired exclusive global rights to develop, manufacture, and commercialize a second-generation gene therapy for spinal muscular atrophy (SMA). The company had completed preclinical studies of the investigational gene therapy in collaboration with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Chan…
BraunAbility has partnered with the nonprofit organization Laughing at My Nightmare (LAMN) to donate a wheelchair-accessible vehicle to a 6-year-old girl with spinal muscular atrophy (SMA). Fynlee Armstrong, a “sassy and spunky little girl” from Clyde, Texas, requires a wheelchair to move around. Over the last few years,…
Biogen is collaborating with Alcyone Therapeutics to develop an implantable medical device that more easily delivers antisense oligonucleotide (ASO) therapies to the spinal canals of people with certain neurological disorders. Under the terms of the agreement, Biogen will acquire exclusive global rights to Alcyone’s ThecaFlex DRx system in…
