After years of setbacks, biotechs are advancing treatments for Huntington’s disease, offering new hope as clinical studies ...

Kaye, who has held positions at Genzyme and Sarepta Therapeutics — where he led development of the Duchenne muscular ...

ReviR Therapeutics, a pioneering biotechnology company focused on developing novel small molecule RNA modulators for neurogenetic diseases, announced a groundbreaking research grant from the Kennedy's ...

The scientific advancements of the past 30 years have transformed our understanding of the potential of gene ... with rare diseases still do not have access to the right care or treatment.

Oct. 28, 2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing function. These preclinical ...

Researchers have developed the most comprehensive atlas linking plasma proteins to diseases and traits in over 53,000 ...

Rznomics, Inc. announced to secure its expanded access program (EAP) from the United States Food and Drug Administration (FDA) for RZ-001, RNA editing gene therapy product for the ...

The world’s first joint research program in evolutionary medical genomics has been launched in Barcelona, under the direction ...

Last May, a 12-year-old with sickle cell anemia was the first person to receive a new gene therapy to treat the disease. The process is painful, expensive, and still frightening and uncertain ...

People affected by a rare disease suffer from misdiagnosis, a lack of effective care and often no treatment at all ... As many rare diseases are genetic, genome sequencing helps increase ...

Mar. 15, 2024 — Scientists uncovered an overlooked mechanism in the gut immune system of patients suffering from severe cases of Crohn's disease. The discovery may help define how to treat ...