NPR spotlights zatolmilast, a promising investigational drug to treat Fragile X syndrome. Families report life-changing improvements in learning and independence.
British startup Healx has secured FDA approval for a phase 2a clinical trial of an AI-discovered compound that could help manage the symptoms of the genetic disorder Fragile X syndrome. The start of the trial marks another milestone in the use of artificial intelligence to help find new applications for existing drugs by mining patient records and research databases.
In recent weeks, USA Today spent days with FRAXA co-founders Dr. Mike Tranfaglia and Katie Clapp, and their son Andy. Andy lives with Fragile X syndrome and is the focus of a segment of USA Today’s occasional series that explores how scientific advances are transforming care for rare diseases.
Katie Clapp and Michael Tranfaglia’s son was born with a genetic disorder that affects brain development. It makes it hard to learn language and basic daily tasks and often is accompanied by a host of other disorders. To help find a cure, they started a foundation and raised research money. After se…
We haven’t found a cure yet, but we’ve moved closer to understanding fragile X syndrome and identifying treatment targets. We hope this will help our son and other families dealing with this life-changing condition.
Propelled by genome sequencing and social media, thousands of charities have sprung up to finance, coordinate and oversee research for cures. Katie Clapp and her son, Andy, who has Fragile X, a disease that causes intellectual disability, with a therapy horse at Gateway Farm in Merrimac, Mass. Ms. Clapp helped form a group that has spent millions on research for a cure.
Fragile X is rare and not as highly publicized as many other better-known genetic diseases that attract media interest and generate richer revenue streams of giving. The world of the ailing doesn’t prioritize. There is no Find Help 101 manual for funding charities or what makes the public wake up one day and pour out its heart, empty its wallet, join a bike-a-thon for its cure.
There is no effective treatment for the rare genetic disorder Fragile X syndrome, so two parents created a foundation to fund research. But they found there’s no easy road to a cure. For a few weeks last year, Michael Tranfaglia and Katie Clapp saw a remarkable change in their son, Andy…
An experimental drug that helps people who have Fragile X syndrome is raising hopes of a treatment for autism. The drug, called arbaclofen, made people with Fragile X syndrome less likely to avoid social interactions, according to a newly published study. Researchers suspect it might do the same for people with autism.
