Since he's homeschooled due to his physical limitations, 7-year-old Vinny misses out on many childhood experiences — like Valentine's Day parties at school.

Residents came together to deliver thousands of cards, gifts, and thoughtful notes for one very special little boy.

Emergence of novel therapies and pharmacological treatments is driving the growth of the cone rod dystrophy treatment market. CRD is a rare inherited disease, wherein gradual loss of vision is ...

President Trump signed an executive order on Tuesday that would restrict access to treatments, including puberty blockers and hormones, for transgender children and teens. Trump is directing ...

has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.

The evidence surrounding the use of puberty blockers and cross sex hormones in children and teens identifying ... and caregivers,” they wrote. “Treatment decisions should consider the lack ...

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic dystrophy type ... Agency for the treatment of DM1, a monogenic ...

There is no cure for spinal muscular atrophy, but treatments can stop its progression and often lead to some improvement. However, Manitoba has refused to pay for the hugely expensive treatment ...

When children with obesity undergo weight-loss treatment, the results have effects later in life, and the risk of serious health problems and premature death is lower as they reach young adulthood.

The treatment studied involved support for children with obesity and their families designed to motivate healthy diets, exercise and sleep habits -- what is known as "behavioural lifestyle therapy." ...