Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with...
Genetic Analysis of Families Finds Novel Mutations in SMA
Genetic analysis of 28 families with a member affected by spinal muscular atrophy (SMA) identified novel mutations in the SMN1 gene and different genes widely prevalent in SMA patients not found in unaffected family members, a study reveals. The study,...
Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show
Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal muscular atrophy (SMA) types 2 and 3 who were unable to walk, according to final results from an extension Phase 2 clinical trial....
Gene Therapy Given Directly to Spinal Canal Might Be Safer With ‘Silencing’ Step
By taking advantage of a natural process of gene silencing, a new gene therapy approach appears to prevent the toxicity to dorsal root ganglion (DRG) — a specific cluster of sensory neurons — seen in non-human primates during gene therapy studies for neurological...HCP — SMA News Today
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