This page tracks new and upcoming treatment options for spinal muscular atrophy (SMA). What’s in development? What’s promising? What’s available soon? Current topics include gene therapy, onasemnogene abeparvovec (Zolgensma), AAV9-SMN, RG7916, LMI070/NVS-101 (branaplam), SMN1, stem cells, nusinersen, antisense oligonucleotides (ASOs), Spinraza, hnRNP F/H proteins, and more. Jump to section: News, Research, Clinical Trials
Last updated: 3 years ago
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News
- Researchers Outline Promising Future for SMA, DMD With Emergence of Gene Therapies, ASOs Sep 12, 2020
- Improved Treatment Options for SMA Present New Opportunities, Challenges Sep 5, 2020
- Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of Spinal Muscular Atrophy Aug 13, 2020
- B.C. baby randomly selected by drug company to receive life-changing $3 million treatment for spinal muscular atrophy (SMA) Aug 13, 2020
- FDA Approves Oral Treatment for Spinal Muscular Atrophy Aug 7, 2020
- Catalent Gene Therapy Facility Receives FDA Approval as a Manufacturing Site for AveXis’ SMA Gene Therapy Aug 6, 2020
- Study: Patients with Pre-Symptomatic Spinal Muscular Atrophy Demonstrate Sustained Benefit from Nusinersen Jun 23, 2020
- New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA® (nusinersen) Continue to Demonstrate Sustained Benefit From Treatment Jun 10, 2020
- Cure SMA Announces New Funding For Network Of Prominent SMA Clinical Research And Treatment Centers Jun 4, 2020
- FDA grants Orphan Drug status for Shift’s E1V1.11 drug compound to treat all forms of spinal muscular atrophy (SMA) May 14, 2020
- Roche provides regulatory update on risdiplam (RG7916) for the treatment of spinal muscular atrophy (SMA) Apr 7, 2020
- Should Biogen’s Spinraza for SMA be delayed amid COVID-19? No, say these parents Mar 26, 2020
Research
- SMN-primed ribosomes modulate the translation of transcripts related to spinal muscular atrophy Sep 21, 2020
- Genetic modifiers ameliorate endocytic and neuromuscular defects in a model of spinal muscular atrophy: hnRNP F/H proteins could be potential targets for therapy development Sep 16, 2020
- Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4 Sep 15, 2020
- Oral Drug Approved for Spinal Muscular Atrophy Sep 15, 2020
- Treating adults with spinal muscular atrophy with nusinersen Sep 11, 2020
- Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy Sep 4, 2020
- The Ubiquitin Proteasome System in Neuromuscular Disorders: Moving Beyond Movement Sep 3, 2020
- Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes Aug 26, 2020
- Gene therapy for spinal muscular atrophy: Solomon’s consensus in Covid times Aug 20, 2020
- Widespread tissue hypoxia dysregulates cell and metabolic pathways in SMA Aug 13, 2020
- Recent advances in phenotypic drug discovery Aug 7, 2020
- Molecular pathogenesis of spinal bulbar muscular atrophy (Kennedy’s disease) and avenues for treatment Aug 6, 2020
- The Identification of Novel Biomarkers Is Required to Improve Adult SMA Patient Stratification, Diagnosis and Treatment Jul 29, 2020
- Combination molecular therapies for type 1 spinal muscular atrophy Jul 25, 2020
- Incorporating Spinal Muscular Atrophy Analysis by Next-Generation Sequencing into a Comprehensive Multigene Panel for Neuromuscular Disorders Jul 23, 2020
- Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders Jul 15, 2020
- New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges Jul 13, 2020
- Identification of two novel SMN1 point mutations associated with a very severe SMA-I phenotype Jul 10, 2020
- Altered bone development with impaired cartilage formation precedes neuromuscular symptoms in Spinal Muscular Atrophy (SMA) Jul 9, 2020
- Linking epigenetic dysregulation, mitochondrial impairment, and metabolic dysfunction in SBMA motor neurons Jul 9, 2020
- Modern approaches in gene therapy of motor neuron diseases such as spinal muscular atrophy (SMA) Jul 8, 2020
- Whole-blood dysregulation of actin-cytoskeleton pathway in adult spinal muscular atrophy patients: new insights and potential therapeutic targets for SMA patients with longstanding denervation and secondary musculoskeletal pathophysiology Jun 17, 2020
- Characteristics of circular RNAs generated by human Survival Motor Neuron genes: diagnostic and therapeutic purposes in spinal muscular atrophy Jun 15, 2020
- RNA in spinal muscular atrophy: therapeutic implications of targeting Jun 13, 2020
- Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent ‘value for money’ for the NHS? A rapid inquiry into suggestions that it may be cost-effective Jun 11, 2020
- Intragenic and structural variation in the SMN locus and clinical variability in spinal muscular atrophy Jun 8, 2020
- Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy Mar 25, 2020
- Gene-based therapies of spinal muscular atrophy: a piece of history of medicine Mar 4, 2020
- Combinatorial treatment for spinal muscular atrophy Feb 14, 2020
- Spinal muscular atrophy — new therapies, new challenges Jan 10, 2020
- Antisense oligonucleotides in SMA: lessons learned and literature data Dec 20, 2019
- Combined treatment with the histone deacetylase inhibitor LBH589 and a splice‐switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells Dec 6, 2019
- Spinal Muscular Atrophy Modeling and Treatment Advances by Induced Pluripotent Stem Cells Studies Aug 9, 2019
- Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis Feb 22, 2019
Clinical Trials
- Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Sep 10, 2020
- Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi Aug 7, 2020
- An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) Jul 28, 2020
- Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients [Completed] Jul 28, 2020
- Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy Jul 22, 2020
- A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants Jul 22, 2020
- A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy Jul 21, 2020
- Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Jun 29, 2020
- Single-Dose Gene Replacement Therapy for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion Apr 28, 2020
- Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy [Suspended] Nov 8, 2019
