Duchenne muscular dystrophy (DMD) is a devastating genetic disorder that progressively deteriorates muscle function, posing a major challenge for patients and their families. Recently, a promising breakthrough has emerged in the form of Solid Biosciences' experimental gene therapy, SGT-003,
In recent developments, a collaborative research team led by the University of Arizona College of Medicine – Tucson has discovered groundbreaking potential for an FDA-approved osteoporosis drug in the treatment of a rare genetic heart disease. The heart condition in question is dilated c
Imagine unlocking a code that holds the secret to the myriad complexities of brain evolution and the onset of neurological diseases. This concept has come closer to reality with a pivotal study led by a Belgian research team, which delves deep into the genetic regulatory codes that control brain
Huntington’s disease (HD), a devastating inherited neurodegenerative disorder, typically manifests in adulthood, progressively impairing motor functions, cognitive capabilities, and overall brain function over approximately 15 to 20 years post-diagnosis. Despite the discovery over three decades ago
In a significant step toward understanding and combating childhood cancers and congenital disorders, the Gabriella Miller Kids First Pediatric Research Program (Kids First), an initiative of the National Institutes of Health (NIH), has released two comprehensive new datasets. Focusing on childhood
Sangamo Therapeutics, Inc., a leading genomic medicine company, has reported promising updated data from the Phase 1/2 STAAR study. This study explores the efficacy of isaralgagene civaparvovec (ST-920) as a treatment for Fabry disease. The analysis, which reflects data up to September 12, 2024,
