December 3rd 2020, 3:59pm
Treatment with risdiplam may not elicit ophthalmologic toxicity in older patients, as well as infants with immature retinas.
November 28th 2020, 3:00pm
Researchers have published their postmortem report on a case of spinal muscular atrophy type 3 complicated by superficial siderosis, offering details on their neuropathological and molecular findings.
November 26th 2020, 5:45pm
The compiled results revealed that hepatotoxicity related to the gene therapy typically presented as cholestatic, which could be prophylactically treated with prednisolone.
November 21st 2020, 1:30pm
Using DIXON, T2 mapping, and diffusion tensor imaging, the researchers obtained quantitative MRI for 31 patients with spinal muscular atrophy (SMA) types 2 and 3.
November 18th 2020, 10:17pm
Following 6 injections of nusinersen, respiratory muscle performance was significantly better among the treated patients compared with age‐matched historical controls.
November 14th 2020, 11:00am
At the time of treatment, there was just 1 paper documenting the use of nusinersen in a case of more mild type 0 spinal muscular atrophy(SMA).
November 12th 2020, 5:00pm
Researchers have identified additional mutations in both SMN1 and other genes, some of which may be associated with the onset of spinal muscular atrophy (SMA).
November 7th 2020, 1:00pm
The study emphasizes the importance of school support and other psychosocial interventions considering specific deficits across adaptive skills in patients with neuromuscular diseases (NMDs).
November 6th 2020, 3:30pm
The researchers of the review discuss the reported impact of novel therapies for spinal muscular atrophy (SMA) and highlight the need for long-term data and monitoring of respiratory outcomes.
October 31st 2020, 11:00am
Jill Jarecki, PhD, chief scientific officer at Cure SMA explained the current data and potential for gender to impact the severity spinal muscular atrophy (SMA).
