Sarepta Therapeutics, Inc. , the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene ...

This often includes Huntington's disease, Familial Prion disease and Familial Alzheimer's disease, with the latter usually ...

A specialist has shed light on whether cognitive decline can be hereditary, at a time when over 944,000 people are living ...

Dr Kellyn Lee, a chartered psychologist, has dedicated her career to researching dementia, ageing and mental health.

Creutzfeldt-Jakob disease (CJD) is a rare and fatal condition that affects the human brain, causing brain damage that worsens ...

West Nile Virus starts off with a flu-like illness with body aches and vomiting and can lead to fatal meningitis ...

Find out if gene therapy has also find its way into skin diseases. With two approvals in the last 2 years, is it the start of ...

The spokesperson also said that the former president had never been diagnosed with prostate cancer before last week.

Travere Therapeutics stock did suffer a bit of setback last week as the FDA moved back the anticipated decision date on a ...

The FDA recently granted orphan drug and rare pediatric disease designations to Infinant Health for a live biotherapeutic ...

Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases ...

Durham-based Atsena Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for ATSN-201, its gene therapy product for the ...