Author Archives: Patricia Inacio PhD

Raft Digital Therapeutics has launched a demo version of Cloud Bazaar, a therapeutic video game that promotes physical activity and exercise in children with spinal muscular atrophy (SMA). Developed in partnership with Biogen Canada, the game aims to promote exercise adherence by turning physical activities into games…

PerkinElmer‘s EONIS system has been approved in the U.S. to be used by certified laboratories to simultaneously test newborns for spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID). The approval by the U.S. Food and Drug Administration (FDA) makes EONIS the first authorized test to…

Spinraza (nusinersen) continues to show sustained efficacy in improving motor function in infants and toddlers with spinal muscular atrophy (SMA), according to newly released interim data from the SHINE study. The greatest benefits were among children who began being treated with Spinraza before they were 10 months old.

Newborn screening for spinal muscular atrophy (SMA) leads to better outcomes, with babies being diagnosed earlier and with prompt access to life-saving treatments, according to three-year data from the SMA screening program in the state of New York. Gene therapy was the main treatment strategy, and delays in…

Unlike in Australia, the U.S., and even several EU countries, there have been no programs for newborn screening for spinal muscular atrophy (SMA) in the United Kingdom — despite compelling evidence that an early diagnosis can improve patient outcomes. Now, Oxford University in the U.K. has launched a pilot study…

Biohaven Pharmaceuticals has entered into a worldwide licensing agreement to develop and commercialize Bristol Myers Squibb’s taldefgrobep alfa, a Phase 3-ready candidate therapy for spinal muscular atrophy (SMA). The company is planning to launch a Phase 3 trial to test the therapy (also known as BMS-986089) in…

Roche and its subsidiary Genentech are launching a Phase 2/3 clinical trial to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody, in combination with Evrysdi (risdiplam) in children with spinal muscular atrophy (SMA). The MANATEE trial has two parts. In part one, 36…

[Editor’s note: An earlier version of this story did not specify in its opening statement that Spinraza’s use in this proposed trial would follow Evrysdi’s use.] Biogen plans to launch a Phase 3b trial into the safety and efficacy of a higher dose of Spinraza (nusinersen) in children,…

A branch of the Canadian Agency for Drugs and Technologies in Health (CADTH) is recommending that Evrysdi (risdiplam) be added to public health plans across Canada, making it available to spinal muscular atrophy (SMA) patients at low or no cost between 2 months and 25 years of age, provided…

People of Indigenous descent in Canada seem to have the highest prevalence of spinal and bulbar muscular atrophy (SBMA) of any populations in the world, according to a new study. Compared to an SBMA prevalence of one to two per 100,000 in the general population, it’s 14.7 per 100,000 people…