C57BL/6 mouse PK. Animals were dosed iv at 1 mg/kg; po at 5 mg/kg, n = 2; 10% 0.1 N HCl, 10% PG, 25 (20%) Solutol, 100 mM pH 5 citrate buffer formulation.

Spinal muscle atrophy is a genetic disorder. Most forms of SMA are caused by mutations of the survival motor neuron 1 gene (SMN1) on the fifth chromosome, resulting in insufficient expression levels ...

While effective treatment is lengthening life and improving its quality, children with spinal muscular atrophy (SMA) type 1 often have abnormal spinal curvatures like scoliosis that require long-term ...

SEREMBAN: The Negri Sembilan palace has revoked the Datukship conferred on former Universiti Kebangsaan Malaysia (UKM) vice chancellor Prof Dr Mohd Ekhwan Toriman. State secretary Datuk Mohd Zafir ...

There appears to be a high incidence of spinal asymmetry in pediatric patients with type 1 spinal muscular atrophy (SMA) receiving nusinersen, according to a recently published study in the journal ...

Copyright 2025 The Associated Press. All Rights Reserved. Copyright 2025 The Associated Press. All Rights Reserved. In a part of downtown Atlanta, shipping containers ...

The story, ‘Nafisa’s tale: Living with SMA type 1’ was the first time her parents spoke to Tanzanian media in efforts to seek community help to get their daughter a life-saving treatment. SMA is a ...

1 Australian Institute of Marine Science, Townsville, QLD, Australia 2 Australian Institute of Marine Science, Indian Ocean Marine Research Centre, University of Western Australia, Crawley, WA, ...

EXG001-307 is a next generation recombinant adeno-associated virus (rAAV) gene therapy in development for Spinal Muscular Atrophy (SMA) Type 1. Data from nine patients demonstrate improved head ...

Exegenesis Bio is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 Phase 1/2 clinical trial in Spinal Muscular Atrophy (SMA) Type I at the American Society ...

1Canada's Michael Smith Genome Sciences Centre, BC Cancer Research Institute, Vancouver, British Columbia, Canada. 2Department of Molecular Oncology, BC Cancer Research Institute, Vancouver, British ...