Motor Gains Best With Early Use of Spinraza for Likely Type 1 Infants
Spinraza (nusinersen) continues to show sustained efficacy in improving motor function in infants and toddlers with spinal muscular…
Articles by Patricia Inacio, PhD
SMA Newborn Screening Improves Outcomes in New York State Data
Newborn screening for spinal muscular atrophy (SMA) leads to better outcomes, with babies being diagnosed earlier and with prompt…
First Newborn Screening for SMA in UK Launched in Oxford Pilot Study
Unlike in Australia, the U.S., and even several EU countries, there have been no programs for newborn screening for…
Biohaven Plans Phase 3 Trial for Therapy After Buying Global Rights
Biohaven Pharmaceuticals has entered into a worldwide licensing agreement to develop and commercialize Bristol Myers Squibb’s taldefgrobep alfa,…
Pediatric Phase 2/3 Trial to Test Anti-myostatin Antibody with Evrysdi
Roche and its subsidiary Genentech are launching a Phase 2/3 clinical trial to evaluate the safety and efficacy…
Trial of Spinraza at High Dose in SMA Patients Who Used Evrysdi Planned
[Editor’s note: An earlier version of this story did not specify in its opening statement that Spinraza’s use in this…
Evrysdi Favored for Public Health Plans in Canada, Within Limits
A branch of the Canadian Agency for Drugs and Technologies in Health (CADTH) is recommending that Evrysdi (risdiplam) be added…
Indigenous Groups in Canada May Have Highest Rate of SBMA in World
People of Indigenous descent in Canada seem to have the highest prevalence of spinal and bulbar muscular atrophy (SBMA) of…
Some SMA Patients Unable to Walk Unassisted May Have Trouble Chewing
Difficulties with biting and fatigue while chewing are common among patients with type 2 and type 3 spinal muscular…
Texas Adds SMA to Its Newborn Screening Program
Newborn screening for spinal muscular atrophy (SMA) is now offered to 85% of all annual births in the U.S., Cure…