44 documents

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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...
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00:54 , Feb 24, 2018 |  BioCentury  |  Regulation

Herculean task for DMD

Driven by the delayed reimbursement decision for Europe’s only approved DMD therapy, the patient advocacy group Duchenne UK has brought together seven companies in the space to create a set of shared tools that should...
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22:30 , Feb 2, 2018 |  BioCentury  |  Product Development

AA Viral meme

Gene therapy pioneer James Wilson’s revelation of two new types of toxicity in animals receiving high systemic doses of adeno-associated viral vectors is unlikely to herald a major disruption of the AAV space, though it...
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23:55 , Jan 4, 2018 |  BC Extra  |  Financial News

SomaLogic completes $200M financing round

Protein analysis company SomaLogic Inc. (Boulder, Colo.) said it completed a $200 million tranched financing round led by iCarbonX. Nan Fung Life Sciences and Madryn Asset Management also participated. SomaLogic's SOMAscan technology can measure 5,000 proteins...
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01:29 , Dec 23, 2017 |  BioCentury  |  Regulation

Prioritizing preferences

The realization that patients have distinct preferences about the trade-offs and risks inherent in medical decisions -- and that their choices are frequently different from those physicians or regulators would make -- has mobilized FDA,...
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20:22 , Nov 3, 2017 |  BioCentury  |  Regulation

Frame work

A little over four years into its implementation, FDA’s structured benefit-risk framework appears to be meeting its objectives of creating transparency into its approval decisions. The agency began to incorporate the framework templates into its reviews...
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19:11 , Oct 13, 2017 |  BioCentury  |  Regulation

Seeking surrogates

Thanks to flaws in PTC Therapeutics Inc.’s trials, the recent FDA advisory committee meeting to discuss Translarna ataluren did nothing to clarify what level of dystrophin expression would be necessary to support an accelerated approval...
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18:01 , Sep 1, 2017 |  BC Week In Review  |  Clinical News

Catabasis' edasalonexent misses primary in Phase II for DMD

In April, Catabasis Pharmaceuticals Inc. (NASDAQ:CATB) reported data from the Phase II portion of the U.S. Phase I/II MoveDMD trial in patients ages 4-7 with Duchenne muscular dystrophy (DMD) not on corticosteroids showing that edasalonexent...
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20:23 , Jul 28, 2017 |  BC Week In Review  |  Clinical News

FDA to allow limb function endpoint in Capricor's DMD trials

Capricor Therapeutics Inc. (NASDAQ:CAPR) said it received minutes from an FDA meeting indicating the agency’s “willingness to accept" the Performance of the Upper Limb (PUL) outcome measure as a BLA-supportive primary efficacy endpoint in trials...
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00:10 , Jul 28, 2017 |  BC Extra  |  Clinical News

FDA to allow limb function endpoint in Capricor's DMD trials

Capricor Therapeutics Inc. (NASDAQ:CAPR) jumped $0.29 (29%) to $1.30 on Thursday after saying it received minutes from an FDA meeting indicating the agency’s “willingness to accept" the Performance of the Upper Limb (PUL) outcome measure...
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