Kasey Mumford, 24, says it took 23 appointments before doctors diagnosed her newborn son, Chester, with a rare condition ...

According to the National Institute of Neurological Disorders and Stroke, there are currently only three FDA-approved ...

Emma, a third-grader with big plans for her future, received a groundbreaking treatment at just the right time.

The death of Raymond Harper, a.k.a. Rolling Ray, has been revealed to be due to natural causes. As previously reported, Ray, ...

Columnist Alyssa Silva shares how a stranger's story helped her parents navigate her SMA diagnosis — a testament to the power ...

A Glasgow man says he is “proud” that Scotland will begin screening new born babies for a rare genetic condition.

A new international study has shown that treating babies with spinal muscular atrophy before symptoms appear can dramatically improve outcomes.Emily ...

The FDA in September issued two rejections for spinal muscular atrophy therapies—both linked to manufacturing problems—and ...

The cause of death of social media star Rolling Ray has been revealed, and according to the Maryland Office of the Chief Medical Examiner, he died of natural causes. Raymond Harper, the ...

Mum-of-two Kasey Mumford says it took 23 appointments before doctors diagnosed her newborn son, Chester, with a rare ...

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...

Not sure how to bring up a treatment change with your SMA doctor? Learn communication tips, key questions to ask, and a ...