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Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type 3 and to be safe in non-human primates, supporting its move into clinical testing. Genecradle Therapeutics is…
Gene therapy that selectively delivers the SMN1 gene to the liver reduced alterations related to spinal muscular atrophy (SMA) in a mouse model, restoring muscle size and correcting pancreatic issues. Mice that received liver-specific SMN1 also lived longer, performed…
A combination genetic therapy approach designed to restore more normal activity of the SMN1Â gene improved motor function and prolonged survival in a spinal muscular atrophy (SMA) mouse model. Called Gene-DUET, it involves supplementing the body with additional healthy SMN1Â genetic material, an approach similar to the approved gene therapy…
Use of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was generally well tolerated and led to improvements in motor function for most children with spinal muscular atrophy (SMA) who were treated at centers across Brazil. “This is the first Brazilian cohort to demonstrate the benefits of [gene therapy]…
Jerry R. Mendell, MD, has been named to the inaugural TIME100 Health list for his decades-long contributions to the treatment of neuromuscular diseases — such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) — and the advancement of gene therapy. The global media brand’s…
SMN-K186R, a modified version of the SMN protein that is deficient in people with spinal muscular atrophy (SMA), was more effective than normal SMN when delivered by gene therapy, a mouse study demonstrated. Treatment with lower doses of the modified SMN gene therapy, to reduce liver-related toxicity…
In children with spinal muscular atrophy (SMA) who have a less-than-optimal response to the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), subsequent treatment with Spinraza (nusinersen) can improve motor function and may reduce nerve damage. That’s according to interim data from the Phase 4 RESPOND study…
Despite concerns about long-term efficacy and safety, gene therapy was the first choice treatment for most parents of children with spinal muscular atrophy (SMA), whether diagnosed via newborn genetic screening or symptoms, according to a new survey study. However, the amount and type of information parents received upon…
Voyager Therapeutics is collaborating with Novartis to discover and develop next-generation gene therapies for conditions like spinal muscular atrophy (SMA). Under terms of the agreement, Novartis will obtain a target-exclusive license to Voyager’s TRACER capsids for SMA and Huntington’s disease, and it will…
Blood levels of troponin-I, a marker for heart muscle damage, are best measured before and after gene therapy in each newborn with spinal muscular atrophy (SMA), a new study concluded. Although elevated troponin-I has been associated with SMA gene therapy, levels above the reference values generated from adults…
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