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

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

Research reveals that loneliness is linked to numerous diseases observationally, but genetic analysis shows it's likely a ...

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 ...

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

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

While most gene therapy research has focused on rare disease, new results are out for a possible treatment for wet-AMD, the ...

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

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

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

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