NICE Recommends Funding of Spinraza in England’s National Health System for all SMA Patients
Patients of all ages with spinal muscular atrophy (SMA) will now be able to be treated with Spinraza (nusinersen) in England, after the…
News
‘I’ve Lived Through Death’ with SMA and ‘Through Life’ with Spinraza, Mom Says in Interview
Destiny McCalvin gave birth to her second child, a girl she named Kenzlee, after what she and her doctors took to be an uneventful pregnancy in 2011. “She seemed like a normal baby; she weighed 7 pounds 7 ounces, no issues,” her mother said. But “issues” soon became clear: “It…
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…
#AANAM – Spinraza Safe and Effective in Adults with SMA, Studies Suggest
Treating adults with spinal muscular atrophy (SMA) with Spinraza (nusinersen) was safe and improved their muscle strength, and motor and respiratory function, among other benefits, according to preliminary research. While treatment with Biogen‘s Spinraza has been demonstrated to be beneficial in children and infants with SMA,…
#AANAM – Risdiplam Shows Promise for Treating SMA Types 2 and 3, Clinical Data Shows
Risdiplam continues to show promise as a therapy for individuals with spinal muscular atrophy (SMA) types 2 and 3, results from an ongoing Phase 2/3 trial show. Results from the study, “Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including…
#AANAM – Risdiplam Improves Survival, Motor Function in Infants with Type 1 SMA, According to Data
Risdiplam (RG7916) can improve both survival and motor function in patients with type 1 spinal muscular atrophy (SMA) beyond that expected in the natural history of the disease, according to recent clinical data. These data were presented during the 2019 annual meeting of the American Academy of…
#AANAM – Zolgensma Continues to Extend Survival in SMA Type 1 Patients Several Years After Dosing
A single administration of Zolgensma continues to extend the survival of patients with spinal muscular atrophy (SMA) type 1 while halting motor neuron loss several years after dosing, according to long-term clinical data. Zolgensma, developed by AveXis — later purchased by Novartis in 2018 — is a one-time treatment intended…
#AANAM – Interim Results from Phase 2 Trial of Spinraza in Infants with SMA Support Efficacy, Safety
Spinal muscular atrophy (SMA) treatment with Biogen’s Spinraza (nusinersen) has resulted in motor milestone achievements in line with normal motor development and continues to provide benefit to infants who began receiving the treatment in a presymptomatic stage of the disease, according to interim clinical trial results. The study, “…
#AANAM – Spinraza Continues to Demonstrate Safety and Provide Improvements in SMA Patients
Treatment with Spinraza (nusinersen) results in greater improvements in motor function and continues to improve or stabilize motor function scores in spinal muscular atrophy (SMA) patients, according to recent clinical data. The findings were presented during the 2019 American Academy of Neurology (AAN) Annual Meeting, being…
#AANAM – Rapid Motor Function Improvement with Zolgensma in Presymptomatic Infants with SMA Types 1-3, Early Trial Results Show
Treatment of presymptomatic babies with the investigational gene therapy Zolgensma provides rapid improvement in motor function in infants with spinal muscular atrophy (SMA) types 1, 2 and 3, according to preliminary results of a Phase 3 trial. The study, named SPR1NT (NCT03505099), is enrolling participants…
