Testing GLP

NoraVolkow(right)saidinaconversationwithSTAT'sElaineChen:“Thisisastructuralproblemthatwehave:Thatwe’ Screenshotviacallingbullshit.orgTiredofalternativefacts,fakenews,andbreathlesshyperbole,twoprofessor




comprehensive

author：Wikipedia    Page View：4113
The FDA building -- First Opinion coverage from STAT
Adobe

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons.

The medicine, marketed as Lenmeldy, is the first treatment for MLD, which is otherwise fatal. Lenmeldy has to be given before symptoms appear or, in some cases, after the first signs of disease, so it won’t be helpful for many patients living with MLD today. But when given in time, it appears to be near-curative, allowing some children to grow up and attend school with unaffected peers.

advertisement

Lenmeldy is already marketed in Europe, under the name Libmeldy, and has helped a handful of patients in the U.S. who were treated under compassionate use, as their doctors feared they didn’t have time to wait for approval.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In
Previous article： Kellogg’s is going to war over Mexico’s nutrition label rules
Next article： Virginia high school admissions case could be legal follow