Former Little Mix star revealed her twin daughters had been diagnosed with Spinal Muscular Atrophy (SMA) Type 1 ...

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

A study led by Aaron Hobbs, Ph.D., and Rachel Burge, Ph.D., at MUSC Hollings Cancer Center, reveals why a specific gene ...

Medicaid is doing a novel payment system for the new, promising and expensive sickle cell treatment. It may become a model ...

Stem cell IVF could enable same-sex couples and single parents to have genetically related children. It’s time to think ...

Sarepta Therapeutics saw its stock skid 11% after the company announced preliminary unaudited 2025 sales results that missed analyst forecasts for its marketed Elevidys® (delandistrogene ...

Ocugen, Inc. gene therapy outlook: key 2026 catalysts, Phase 2/3 eye-disease data, BLA plans to 2028, and risks. Click for ...

The company made an even more dramatic claim the following month, when it announced it had created three dire wolves. These ...

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...

Brazil holds a record number of supercentenarians, people who live healthy lives past 110. What is the secret to their ...

Two variants in a single gene are implicated in almost all Alzheimer’s and nearly half of all dementia cases, according to ...