23:58 , Jun 21, 2018 |  BC Extra  |  Company News

Sarepta says no to right to try for its DMD therapies

Following data from a small trial of a Duchenne muscular dystrophy gene therapy, Sarepta Therapeutics Inc. (NASDAQ:SRPT) told BioCentury that it will not offer pre-approval access under right-to-try legislation or FDA’s expanded access program for...
21:00 , Jun 7, 2018 |  BC Extra  |  Company News

BrainStorm considering right to try for ALS therapy

BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) is considering offering NurOwn to treat amyotrophic lateral sclerosis under the recently enacted federal right-to-try law, BrainStorm President and CEO Chaim Lebovits told BioCentury. He also discussed the company’s plans...
23:22 , May 17, 2018 |  BC Extra  |  Politics & Policy

House to vote on Senate's right-to-try bill

House Majority Leader Kevin McCarthy (R-Calif.) said the House of Representatives will vote next week on a right-to-try bill ( S. 204 ) passed by the Senate last August that seeks to authorize using investigational...
17:29 , Mar 23, 2018 |  BC Week In Review  |  Company News

NORD grades states on health policy

The National Organization for Rare Disorders said in its annual report card that many state laws were not sufficient to reduce the burden of rare disease on Americans. The report, which grades the strength of...
23:26 , Mar 20, 2018 |  BC Extra  |  Politics & Policy

NORD says states come up short on health policy

The National Organization for Rare Disorders said in its annual report card that many state laws were not sufficient to reduce the burden of rare disease on Americans. The report , which grades the strength...
18:08 , Dec 20, 2017 |  BC Innovations  |  Distillery Techniques

Drug platforms

TECHNOLOGY: Gene therapy An ADARB2-based method of editing RNA could help treat diseases caused by point mutations, such as Rett syndrome and X-linked nephrogenic diabetes insipidus. The method repairs disease-associated G to A mutations in...
17:40 , Oct 12, 2017 |  BC Extra  |  Preclinical News

Team describes MECP2 gene therapy approach for Rett syndrome

In a paper published in...
19:40 , Oct 3, 2017 |  BC Innovations  |  Distillery Therapeutics

Other

INDICATION: Other / Genetic disorders Mouse studies suggest that inhibiting PP2A could treat MECP2 duplication syndrome, an X-linked genetic disorder with neuromotor, developmental and other symptoms. In a mouse model of the syndrome, tool compounds...
01:31 , Sep 2, 2017 |  BioCentury  |  Strategy

Breach of contract

Merck & Co. Inc. Chairman and CEO Kenneth Frazier’s resignation in August from a White House advisory council was an act of principle, and a reminder that 30 years ago Merck was one of the...
00:03 , Aug 24, 2017 |  BC Extra  |  Preclinical News

Inhibiting PP2A reduces MECP2 in neuropsychiatric model

In a paper published in...