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

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 ...

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 ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. Genetic factors are very important in ...

Former England rugby captain Lewis Moody has been diagnosed with motor neurone disease (MND), a progressive condition ...

The apitegromab approval process reminded guest writer Joanna Buoniconti of lessons about keeping hope alive while protecting ...

Now, the family is launching a public campaign to raise $1.5 million to support research into gene therapy that could potentially lead to a cure. The funds will go to the SMA-PME Research Charity, ...

SMA Europe has launched an online tool designed to help patients, doctors, and others in the spinal muscular atrophy (SMA) ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses the role of personalized medicine in spinal muscular atrophy.

Could an anomaly in the developing brain explain motor difficulties occurring decades later in people with rare movement ...

The Rare Disease Therapeutics Market is experiencing an unprecedented wave of innovation, driven by gene therapies, RNA-based ...

A large-scale international study has found that women possess twice as many genetic variants associated with major depression as men, offering new insight into long-observed ...