Fifteen children born with a rare muscle-wasting disease would probably not be alive today if not for an experimental treatment that tweaked their genes shortly after they were born.

The disease, called spinal muscular atrophy, would likely have killed most of them by age two. Amazingly, many of the children can now speak and sit on their own, and a few are even walking—milestones they never would have reached without the treatment. The company behind the drug, AveXis, is seeing the most dramatic results in children who are treated in the first month or two of life.

People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home.

President Donald J. Trump

RIGHT TO TRY: Congress passed the Right to Try Act of 2017, sending a priority bill to President Donald J. Trump for his signature.

PHILADELPHIA (WPVI) -- 

Two school communities are coming together in a fight for life for two little boys who are victims of a rare and ominous disease.

"Everybody sees these happy fun loving kids that are just playful and full of energy but the reality is that's all going to go away and that's probably a year or two from happening," said teacher/mom Emily Lieber.

Friday morning dozens of students from Meredith Elementary School and Neumann Goretti High School gathered for the second annual Hoops for Hunters Research Basketball Scrimmage.

Orphan drugs, which refer to drugs developed to treat rare diseases, are emerging as a new battlefield for global pharmaceutical companies. They have been neglected by the industry because the number of rare disease patients is small and it is hard to carry out clinical trials. However, orphan drugs are expected to become a new niche market as more and more orphan drugs developed by domestic companies are registered with the U.S Food and Drug Administration (FDA).

This morning, REGENXBIO Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to RGX-121, a novel, one-time investigational treatment for mucopolysaccharidosis type II (MPS II).