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England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), according to a press release from SMA UK. NICE had previously planned to conduct evaluations of clinical trial and cost-effectiveness data,…
Treatment combinations, including future muscle-targeted therapies, are part of what likely lies ahead for spinal muscular atrophy (SMA), according to experts in the SMA community. In separate interviews with SMA News Today, Richard Finkel and Laurent…
As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. Baby’s First Test is a program of Expecting Health, an organization focused on pregnancy and newborn health.
SMA Europe has launched an alliance that is demanding that, by 2025, newborn screening programs in Europe include a test for spinal muscular atrophy (SMA). The new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance) was launched as a final initiative to mark…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected…
Increasing the levels of brain-derived neurotrophic factor (BDNF) — a molecule involved in the communication between nerve cells and muscle — specifically in muscle delayed symptom onset and slowed disease progression in a mouse model of spinal and bulbar muscular atrophy (SBMA), a study has found. BDNF’s…
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to SMN1 via CRISPR/Cpf1 and single-stranded oligodeoxynucleotides in…
View this post on Instagram Day 31 of 31 Days SMA Topic: Fashion and SMA This is…
Novartis’ gene therapy Zolgensma is generally safe and effective in promoting motor improvements in children with spinal muscular atrophy (SMA) treated up to age 2, according to a multicenter study. However, “if the children are older and heavier, there is a potential for more liver injury [a known side…
The U.S. Food and Drug Administration (FDA) has given Catalent Biologics approval to aid in manufacturing products for use with AveXis’ spinal muscular atrophy (SMA) gene therapy, Zolgensma, at its Maryland facility. The FDA inspected the company’s commercial-scale gene therapy manufacturing center in June. Catalent and…
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