SMA Type 2

Most individuals with spinal muscular atrophy (SMA) type 2 experience improvements in physical abilities within a year after starting treatment with Spinraza (nusinersen). However, about 1 in 3 patients had some loss of function in the first year after beginning the approved SMA therapy. That’s according to…

Scholar Rock remains on track to report topline results later this year from its ongoing Phase 3 clinical trial of apitegromab in children and young adults with spinal muscular atrophy (SMA) types 2 or 3. Apitegromab is an antibody that targets myostatin, a protein involved in suppressing…

A previously unknown breathing problem — called pseudo-obstructive sleep-disordered breathing — has been discovered among people with spinal muscular atrophy (SMA) type 2, a study reveals. This unusual breathing pattern during sleep is caused by an imbalance between the SMA-related weakness in the chest muscles and the relatively unaffected…

A young woman with spinal muscular atrophy (SMA) type 2 gave birth to a premature baby boy with the help of a multidisciplinary team despite having severe breathing problems, according to a report. The woman’s shortness of breath got worse during pregnancy and she…

The Muscular Dystrophy Association (MDA) has chosen Ira Walker, a Florida resident living with spinal muscular atrophy (SMA) type 2, as this year’s national ambassador. In this new role, the 39-year-old Walker will share his story nationwide, with an aim of raising awareness of the MDA’s mission…