A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most ...

Using technology to deliver therapy for children and young people is as effective as traditional face-to-face therapy, according to new research. The University of East Anglia-led study highlights the ...

A new Hennepin County stabilization center hopes to serve kids with complex mental health needs who otherwise end up in an ER or detention center.

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

A common misconception about rare disorders is that they only effect a small number of people. However, according to the National Organization for Rare Disorders, around 25-30 million Americans are ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Carlos, a six-year-old Hispanic American diagnosed with autism spectrum disorder (ASD), is primarily non-verbal. At age two, he was removed from his home due to abuse and neglect. Carlos and his four ...

Thousands of disabled and autistic children in Florida are losing medically-prescribed therapy after an audit revealed that unqualified therapists were being paid through Medicaid. On Dec. 31, ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...