Stop Squabbling, Mom Tells Troubled Biotech: 'Just Get My Kid the Drug
'Imagine for a harrowing moment that your child has a debilitating, deadly disease for which there’s no cure. A biotech company has a drug that might arrest your child’s decline, maybe keep him out of a wheelchair, possibly add years or decades to his life. It would be tough—no, unbearable—to watch infighting at the company impede access to the medication.
“It’s heartbreaking,” Mindy Leffler told me by phone over the weekend. Her son Aidan, 11, has Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disease that afflicts 1 in 3,500 boys born in the U.S. DMD robs its victims of the ability to walk in early adolescence. Pulmonary or other complications typically kill sufferers in their twenties.
Leffler was reacting to a regulatory filing and related news reports about the firing of a key executive at Sarepta Therapeutics (SRPT), a biotech company in Cambridge, Mass. Sarepta is seeking Food and Drug Administration approval for a DMD treatment that could keep Aidan on his feet and extend his life. On Thursday, Sarepta disclosed that it had fired its chief scientific officer, Arthur Krieg. The company didn’t explain the move.
Stop Squabbling, Mom Tells Troubled Biotech: 'Just Get My Kid the Drug
ReplyDelete'Imagine for a harrowing moment that your child has a debilitating, deadly disease for which there’s no cure. A biotech company has a drug that might arrest your child’s decline, maybe keep him out of a wheelchair, possibly add years or decades to his life. It would be tough—no, unbearable—to watch infighting at the company impede access to the medication.
“It’s heartbreaking,” Mindy Leffler told me by phone over the weekend. Her son Aidan, 11, has Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disease that afflicts 1 in 3,500 boys born in the U.S. DMD robs its victims of the ability to walk in early adolescence. Pulmonary or other complications typically kill sufferers in their twenties.
Leffler was reacting to a regulatory filing and related news reports about the firing of a key executive at Sarepta Therapeutics (SRPT), a biotech company in Cambridge, Mass. Sarepta is seeking Food and Drug Administration approval for a DMD treatment that could keep Aidan on his feet and extend his life. On Thursday, Sarepta disclosed that it had fired its chief scientific officer, Arthur Krieg. The company didn’t explain the move.
http://www.businessweek.com/articles/2014-07-26/biotech-turmoil-threatens-new-muscular-dystrophy-treatment
This is too logical.
ReplyDelete