Phase 2 Trial Evaluating Mestinon, Already Approved for Myasthenia Gravis, for SMA Types 2-4
A Phase 2 clinical trial is testing the effectiveness of Mestinon (pyridostigmine…
Articles by Ana Pena, PhD
Psychological Support Relieves Anxiety of Spinraza Treatment in Kids With SMA Type 1, Study Says
Psychological interventions to control emotional stress can help relieve the anxiety that parents and children with…
New Mutation Linked to SMA-LED in 4 Unrelated Children, Case Study Reports
A newly identified mutation in the DYNC1H1 gene causes a rare form…
Case Report Alerts Physicians About Link Between SMA and Ketoacidosis
Spinal muscular atrophy (SMA) patients can be more prone to a life-threatening metabolic imbalance called ketoacidosis,…
Protein Independent of SMN May Be Worthy Target as Combo Treatment for SMA, Mouse Study Suggests
Lowering the levels of a protein called CHP1, together with Spinraza (nusinersen) treatment, may help to…
New Potential Therapeutic Target for SMA Identified in Mouse Study
A key player in a pathway known as the cell’s “cleaning system” could be a promising target for alternative approaches…
Cell ‘Cleaning System’ Could Be Alternative Target for SMA Therapies, Scientists Say
A pathway known as the cell’s “cleaning system” may be a promising target of alternative approaches to treating…
Nerve Cell Function Impacted When Movement is Limited, Mouse Study Suggests
Neurological health depends as much on signals sent by the body’s leg muscles to the brain as it does on…
AveXis’ SMA Type 1 Gene Therapy Granted Speedier Review in Japan
AVXS-101 has been awarded the SAKIGAKE designation in Japan for the treatment of spinal muscular atrophy (SMA) type…
Why Some Parents of Children with SMA Hesitate to Let Them Participate in Trials
Many parents of children with neuromuscular disorders like SMA would like to see if yet-to-be-approved therapies could help…