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People living with spinal muscular atrophy (SMA) who need financial help to pay for treatment may now apply for aid through a new patient assistance program offered by the PAN Foundation. The foundation’s new program will provide up to $6,500 annually to each eligible patient. The…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than…
Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for spinal muscular atrophy (SMA) patients in Japan. A new drug application seeking approval, submitted to that country’s Ministry of Health, Labour and Welfare (MHLW), may be given priority…
Adults with spinal muscular atrophy (SMA) who began being seen at specialty centers after the approval of Spinraza (nusinersen)Â may have more severe symptoms than adults who have been followed regularly at such centers, a new study suggests. The study, “Sometimes they come back: new…
A new campaign is seeking to raise awareness of key motor development milestones for infants that, if missed, could mean spinal muscular atrophy (SMA) or other neuromuscular conditions. It’s been launched by Novartis Gene Therapies in collaboration with a diverse group of experts in the United Kingdom.
Balance is typically a part of everyone’s treatment plan during physical therapy. Whether you have back pain or ankle pain, improving your balance carries over to an increase in functional mobility that will in turn improve your functional strength and increase your safety, typically assisting to improve the ailment you…
Gene therapy that increased the production of a protein called DOK7 in a spinal muscular atrophy (SMA) mouse model was found to improve the neuromuscular junction structure, reduce muscle fiber loss, improve grip strength, and extend survival. These study findings suggest that…
Adults with spinal muscular atrophy (SMA) type 3 have significantly lower levels of several molecules that help regulate actin, one of the major components of the cell’s cytoskeleton, according to a study. These findings, from blood and connective tissue cells, add to information from a previous study…
Treatment with the molecule 4-aminopyridine (4-AP) failed to improve the motor function, endurance, and muscle strength of spinal muscular atrophy (SMA) type 3 patients who are able to walk, according to results from a small Phase 2/3 clinical trial. Findings from the trial…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest…
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