The FDA is already under fire from lawmakers for recent delays and rejections of new drugs for rare diseases.

Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and ...

Jeff Allen, a $10 million “Beast Games” winner, rucked five marathons in five days across ENC to raise money and awareness ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

A dog owner is raising awareness after her three-year-old Staffordshire Bull Terrier was diagnosed with a rare and incurable ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

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

Subjecting participants to invasive procedures without the prospect of therapeutic benefit is unjustifiable,” says the CEO of ...

One-year-old Bonnie-Mai ...

Research uncovering the origin of pineoblastoma, a rare pediatric brain tumor, has also revealed a dependency across multiple brain tumor types that share a similar molecular program. Scientists at St ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

A team of researchers at Texas Children’s Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new approach to treat Rett ...