Larry Luxner,  —

Move over, Sarepta Therapeutics, Solid Biosciences, PTC Therapeutics and Pfizer. Vertex Pharmaceuticals, which dominates the cystic fibrosis drug market, has just entered the multibillion-dollar race to find effective treatments — and maybe even cures — for two other rare diseases: Duchenne muscular dystrophy…

Years ago, doctors believed that no exercise was best for boys with Duchenne muscular dystrophy (DMD). Today, DMD experts all agree: exercise extends lives. But what kind of exercise is ideal for Duchenne kids? How much is too much? And what about stretching, night splints and specialized equipment? Physical…

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…

Climbing up the world’s tallest mountain isn’t for everyone. With that in mind, a small New England-based charity plans just such an adventure this November — for the third time, no less — to raise at least $100,000 to help cure Duchenne muscular dystrophy (DMD). Tonya Dreher is executive…

Filippo Buccella, an Italian pharmacist from Rome, clearly remembers the day in 1993 when he brought his nearly 3-year-old son, Luca, to the hospital. Luca was about to undergo surgery for his adenoids when a test revealed that his creatine kinase level was over 11,000 — a hallmark symptom of…

If there’s anyone who knows a thing or two about how to discuss Duchenne muscular dystrophy (DMD) with your son, his siblings, his teachers and his friends, it’s James Poysky. A clinical psychologist in Katy, Texas, Poysky teaches at Houston’s Baylor College of Medicine and chairs a…

Back in June 1984, a doctor calmly informed Pat Furlong that her sons — 4-year-old Chris and 6-year-old Patrick — had Duchenne muscular dystrophy. It was the first time she had ever heard those three words uttered together. The disease eventually claimed both boys, but that didn’t stop Furlong’s fight against…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

Now that the U.S. Food and Drug Administration (FDA) has approved Zolgensma — the world’s first gene therapy for spinal muscular atrophy (SMA) — experts say a similar gene therapy to cure Duchenne muscular dystrophy isn’t far behind. On May 24, the FDA gave its long-awaited…