A severe illness would test any family. For the Secklers and Wicka families in the U.S., their sons' development of Duchenne muscular dystrophy has inspired a heroic attempt to develop a new treatment. The Wall Street Journal has the story, which tracks their decision to buy an experimental DMD drug from an Israeli biotech and set up a virtual company under the guidance of an industry veteran.
Dyax vet Marc Blaustein now runs Halo Therapeutics out of his living room. "We want to quickly determine if this is a good drug for Duchenne muscular dystrophy," Blaustein tells the Journal. And they're out raising $8 million to get the treatment through a mid-stage study.
They may face the same tough odds that anyone in biotechnology has to reckon with. But with patient advocacy groups receptive to the idea of helping fund the next stage of development, the two families have a shot at reaching the proof-of-concept stage.
- here's the story from the WSJ