Dementia often affects memory, thinking, behavior and daily functioning — now, a new case study reveals a novel symptom that shouldn’t be overlooked.

The MDA meeting is a key opportunity to highlight the expanding real‑world evidence shaping care for the Duchenne community,” said Will Andrews, MD, Chief Medical Officer of Catalyst. “Through our ...

Gregory and Tara Scott are camping outside a new Buckhead store, hoping to raise money for gene therapy research for their ...

A Carlsbad family is sharing their son’s story after the 5-year-old was diagnosed with a rare genetic disorder and began a ...

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

At age 7, Lukas Caldwell developed a faint discoloration on his face and was diagnosed with Parry-Romberg Syndrome, a rare disorder that gradually destroys facial tissue.

"Beast Games" winner Jeff Allen hopes finding a cure for his son's rare disease will unlock new treatments for other brain conditions.

There is a process to a patient being diagnosed with a rare condition and then collaborating with their physician, if they are fortunate enough to have that opporunity. One recent qualitative study ...

This Ice Age pair of hunter-gatherers were both shorter than average for their population – one of them markedly so – and now we finally know why. A team of researchers from Europe re-examined the ...

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

For too long, the promise of personalized therapies has been tantalizingly close, yet frustratingly out of reach for ...

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...