Unlike in Australia, the U.S., and even several EU countries, there have been no programs for newborn screening for…
Articles by Patricia Inacio, PhD
Biohaven Pharmaceuticals has entered into a worldwide licensing agreement to develop and commercialize Bristol Myers Squibb’s taldefgrobep alfa,…
Roche and its subsidiary Genentech are launching a Phase 2/3 clinical trial to evaluate the safety and efficacy…
[Editor’s note: An earlier version of this story did not specify in its opening statement that Spinraza’s use in this…
A branch of the Canadian Agency for Drugs and Technologies in Health (CADTH) is recommending that Evrysdi (risdiplam) be added…
People of Indigenous descent in Canada seem to have the highest prevalence of spinal and bulbar muscular atrophy (SBMA) of…
Difficulties with biting and fatigue while chewing are common among patients with type 2 and type 3 spinal muscular…
Newborn screening for spinal muscular atrophy (SMA) is now offered to 85% of all annual births in the U.S., Cure…
Evrysdi (risdiplam) has been approved in Japan as an oral, at-home treatment of spinal muscular atrophy (SMA). The flavored liquid therapy…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…