Larry Luxner,  —

Articles by Larry Luxner

Vertex Enters Duchenne Therapy Market with $1B Acquisition of Exonics

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…

As PPMD Turns 25, Founder Pat Furlong Reflects on Advances in Duchenne

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…

European Initiative Targets Diagnosis, Treatment of Rare Diseases

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…

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

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…