A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...

Early research shows a one-time gene therapy has potential for addressing both the liver and lung disease that patients with ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

Here, experts explain genetics in Alzheimer’s and dementia, including if it’s hereditary, how to lower your risk, and ...

In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B ...

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

Initiation of dosing in our Phase 1b clinical trial represents an important step in the clinical development of GT-02287 and in Gain’s mission to deliver a disease-modifying therapy to people with ...

Medically reviewed by Renita White, MD Cancer that develops in the ovaries (the egg-producing reproductive organs) is ...

Faced with an impossible choice after their son's cancer diagnosis, a local family is trying to slow down time. If the odds ...

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President makes rare appearance at the DOJ as Democrats warn against GOP’s ‘dismantling of government’ over shutdown vote ...