[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…
Difficulties with biting and fatigue while chewing are common among patients with type 2 and type 3 spinal muscular atrophy (SMA) who are unable to walk unassisted, a Dutch study reports. The findings highlight the need for interventions to help maintain muscle strength in the jaw of these patients…
Newborn screening for spinal muscular atrophy (SMA) is now offered to 85% of all annual births in the U.S., Cure SMA has announced. The milestone, which means that eight in 10 babies are getting tested at birth, was reached after Texas included the newborn screening on June 1,…
Evrysdi (risdiplam) has been approved in Japan as an oral, at-home treatment of spinal muscular atrophy (SMA). The flavored liquid therapy is also approved in the U.S., Europe, and Canada to treat all types of SMA in patients ages 2 months and older, and its use was favored by regulators…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
Asuragen’s lab test for spinal muscular atrophy (SMA) is as robust and accurate at identifying carriers of the disease and diagnosing patients as more traditional methods, a study shows. The diagnostic test — called AmplideX PCR/CE SMA Plus Kit — was validated independently in four labs and showed over…
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was granted a fast track designation by the U.S. Food and Drug Administration. “We are delighted to receive Fast Track designation and look forward to working closely with the FDA towards our aim of establishing apitegromab as…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
