New report finds 'catastrophic failures' in the current system to support youngsters living with the condition.

A mother is raising $6 million to secure a potential treatment for her 3-year-old daughter with Sanfilippo Syndrome, a rare ...

Researchers at Helmholtz Munich, the Technical University of Munich and the LMU University Hospital Munich uncovered a ...

LOS ANGELES (KABC) -- Most kids set up a lemonade stand to raise money for a new toy or bike, but 4-year-old Emily McGlocklin is raising money to help her sister who is battling a rare disorder. Emily ...

Researchers discovered that a tiny structural feature of the enzyme GPX4 helps keep neurons safe. A rare mutation removes ...

Now Ollie is making history as the world’s first successful gene therapy patient for this cruel condition. Ten months after ...

A study published 30 years ago is striking up new conversation about the potential connection between the common cold sore and Alzheimer’s disease. Published in The Lancet in 1997, researchers ...

Researchers have demonstrated that individuals who had childhood epilepsy have an increased accumulation of brain amyloid later in life, potentially predisposing them to late-onset brain amyloid ...

LOS ANGELES, California -- Most kids set up a lemonade stand to raise money for a new toy or bike, but 4-year-old Emily McGlocklin is raising money to help her sister who is battling a rare disorder.