The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of promising therapies by speeding their evaluation so that the therapies, if proven effective, can be available to patients…
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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
Poland’s Ministry of Health has agreed to pay for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) regardless of age or disease severity — ending a year-long struggle by the country’s nonprofit Fundacja SMA to gain access to one of the world’s most costly drugs. The surprise announcement…
An increased amount of SMN protein — crucial to the survival of motor neurons — in the blood could reflect similar increases in the brain, spinal cord, and peripheral tissues after spinal muscular atrophy (SMA) treatment with the oral therapy risdiplam, researchers say. They believe these findings in animal…
The Cure SMA educational campaign is designed to equip parents and healthcare professionals with the tools needed to recognize motor delays in spinal muscular atrophy (SMA) patients early, allowing infants and children to receive potentially life-saving treatment. Called SMArt Moves, the program cites the fact that Spinraza and…
Later-onset forms of spinal muscular atrophy (SMA) are linked to extensive atrophy in the cervical spinal cord, particularly in gray matter of the vertebrae, that may begin long before symptoms are evident, researchers report. The study, “The spinal and cerebral profile of adult spinal-muscular atrophy: A multimodal imaging study,”…
Researchers at Stanford University School of Medicine found that a molecule called Activating Signal Cointegrator 1 (ASC-1) acts “a central hub” for proteins involved in amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), suggesting that both disorders “are more intimately tied to one another than previously…
Treatment of spinal muscular atrophy (SMA) with Spinraza (nusinersen) through the spinal canal is feasible and leads to high patient compliance as long as it’s done under the supervision of an experienced multidisciplinary team, according to a single-center study in Italy. The study, “Intrathecal nusinersen…
The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma (onasemnogene abeparvovec-xxxx) — previously called AVXS-101 — be approved as a gene therapy for spinal muscular atrophy (SMA) type 1. Its request is reported to specifically cover intravenous…
Administration of Spinraza (nusinersen) directly into the spinal canal is feasible and well-tolerated by adults with spinal muscular atrophy (SMA) type 2 or 3. Still, such treatment can be challenging for SMA type 2 patients and those who have had spinal fusion, a study shows. The study,…
