Rocket Pharmaceuticals announced enrollment completion for phase 2 study using its gene therapy RP-A501 for treatment of male ...

People with rare genetic variants linked to degenerative brain disorders like Parkinson's disease are at increased risk of ...

A research team from Hiroshima University has developed an analysis pipeline to identify unexploited genes for a given ...

The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it ...

It’s been almost a year since the Food and Drug Administration approved the first genetic treatments for sickle cell disease.

Each episode of this journey through a disease state contains both a physician guide and a downloadable/printable patient ...

Jenssen guessed then that two of her daughters, the twins, had a Duchenne mutation, and she knew in her heart that, for at ...

There's new hope for people with sickle cell disease which affects mainly African Americans. It's gene therapy research at ...

Children with spinal muscular atrophy (SMA) experience benefits from receiving a disease-modifying gene therapy, but many ...

Opens in a new tab or window The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the ...

Severe nosebleeds caused by hereditary hemorrhagic telangiectasia dwinded in people who took a drug used to treat cancer.

Mutations in genes can lead to genetic disorders, impacting health. Understand the types, causes, and implications of genetic ...