A Phase 2 clinical trial is testing the effectiveness of Mestinon (pyridostigmine…
Articles by Ana Pena, PhD
Psychological interventions to control emotional stress can help relieve the anxiety that parents and children with…
A newly identified mutation in the DYNC1H1 gene causes a rare form…
Spinal muscular atrophy (SMA) patients can be more prone to a life-threatening metabolic imbalance called ketoacidosis,…
Lowering the levels of a protein called CHP1, together with Spinraza (nusinersen) treatment, may help to…
A key player in a pathway known as the cell’s “cleaning system” could be a promising target for alternative approaches…
A pathway known as the cell’s “cleaning system” may be a promising target of alternative approaches to treating…
Neurological health depends as much on signals sent by the body’s leg muscles to the brain as it does on…
AVXS-101 has been awarded the SAKIGAKE designation in Japan for the treatment of spinal muscular atrophy (SMA) type…
Many parents of children with neuromuscular disorders like SMA would like to see if yet-to-be-approved therapies could help…