Centenary Institute researchers have uncovered new genetic causes of inherited heart conditions, providing families with ...
Scientists at The Hospital for Sick Children (SickKids) have revealed a previously overlooked layer of genetic variation that ...
Outcome of Regenerative Medicine Advanced Therapy (RMAT) meeting provides the potential for an accelerated regulatory pathway ...
Huntington’s disease (HD) is one of those rare conditions that affects not just the patient but the entire family—medically, emotionally and genetically. This inherited brain disorder causes gradual ...
Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...
There are some 80 human diseases that in one way or another are considered autoimmune. Perhaps the most common indication of an autoimmune pathogenesis is the presence of circulating antibodies that ...
Effective management of inherited retinal diseases requires a comprehensive approach to diagnosis and treatment, according to ...
Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.
Liver disease is a complex and multifaceted health concern that affects millions of people worldwide. While lifestyle factors such as alcohol consumption and diet play significant roles in liver ...
Clinical Trials Arena on MSN
Opus Genetics doses first subject in gene therapy trial
Opus Genetics has dosed the first subject in its Phase I/II clinical trial of OPGx-Best1 gene therapy for the treatment of ...
Emerging gene therapy administered through a one-time subretinal injection marks a major milestone for the inherited retinal disease community Initial data expected in Q1 2026 RESEARCH TRIANGLE PARK, ...
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