Move over, Sarepta Therapeutics, Solid Biosciences, PTC Therapeutics and Pfizer. Vertex Pharmaceuticals, which dominates the…
Articles by Larry Luxner
Years ago, doctors believed that no exercise was best for boys with Duchenne muscular dystrophy (DMD). Today, DMD experts…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second…
Climbing up the world’s tallest mountain isn’t for everyone. With that in mind, a small New England-based charity plans just…
Filippo Buccella, an Italian pharmacist from Rome, clearly remembers the day in 1993 when he brought his nearly 3-year-old son,…
If there’s anyone who knows a thing or two about how to discuss Duchenne muscular dystrophy (DMD) with your…
Back in June 1984, a doctor calmly informed Pat Furlong that her sons — 4-year-old Chris and 6-year-old Patrick — had…
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet…
Now that the U.S. Food and Drug Administration (FDA) has approved Zolgensma — the world’s first gene therapy for…