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

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

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

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

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 ...

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

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

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

By Hugo Francisco de Souza While loneliness has been tied to higher disease risks, new genetic findings reveal it might be ...

Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA ...

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