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PTC Therapeutics Announces Agreement to Acquire Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in U.S.

Submitted by KReddy on Thu, 03/16/2017 - 10:38
Emflaza is the first treatment approved in the U.S. for DMD patients 5 years and older, regardless of genetic mutation

Aligns with PTC's mission and commitment to the DMD community

$140 million upfront consideration (comprised of cash and PTC common stock) plus potential contingent payments based on net sales and single milestone payment

PTC to host investor conference call today, March 16, 2017 at 8:30 am ET
MDA Awards $96,360 to Izumi Biosciences

Submitted by KReddy on Fri, 03/10/2017 - 08:22

Investment will support development of ALS drug “enhancer”

CHICAGO, March 10, 2017 – The Muscular Dystrophy Association is pleased to announce the award of an MDA Venture Philanthropy (MVP) grant, totaling $96,360 to Lexington, Mass.-based Izumi Biosciences, Inc., to fund early-stage development of IZ10023, a type of drug called a pharmacokinetic (PK) enhancer,” for use in people with ALS (amyotrophic lateral sclerosis) who are taking riluzole.
MDA Awards More than $7 Million in New Research Grants

Submitted by KReddy on Thu, 03/09/2017 - 07:56

New investment builds on recent breakthroughs with availability of three new neuromuscular disease drugs

CHICAGO, March 9, 2017 – The Muscular Dystrophy Association today announced the award of 29 new research and development grants totaling more than $7 million and targeted to impact the state of the science and accelerate the development of treatments for muscular dystrophy, ALS and related life-threatening diseases.
FDA Accepts sBLA Filing of Soliris® (Eculizumab) as a Potential Treatment for Patients with Refractory Generalized Myasthenia Gravis (gMG)

Submitted by KReddy on Wed, 03/08/2017 - 09:43
PTC Therapeutics Announces FDA Acknowledgment of New Drug Application Filing for Translarna for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

Submitted by KReddy on Wed, 03/08/2017 - 09:39
Eleven Major Patient Groups Outline Joint Position on Health Care Reform

Submitted by KReddy on Mon, 03/06/2017 - 14:46

Washington, D.C., March 6, 2017 – A coalition of eleven nonpartisan patient groups today laid out a joint set of goals they want Congress to focus on as it considers changes to the Affordable Care Act.

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Press Releases

PTC Therapeutics Announces Agreement to Acquire Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in U.S.

MDA Awards $96,360 to Izumi Biosciences

MDA Awards More than $7 Million in New Research Grants

FDA Accepts sBLA Filing of Soliris® (Eculizumab) as a Potential Treatment for Patients with Refractory Generalized Myasthenia Gravis (gMG)

PTC Therapeutics Announces FDA Acknowledgment of New Drug Application Filing for Translarna for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

Eleven Major Patient Groups Outline Joint Position on Health Care Reform

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About Us

Press Releases

PTC Therapeutics Announces Agreement to Acquire Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in U.S.

MDA Awards $96,360 to Izumi Biosciences

MDA Awards More than $7 Million in New Research Grants

FDA Accepts sBLA Filing of Soliris® (Eculizumab) as a Potential Treatment for Patients with Refractory Generalized Myasthenia Gravis (gMG)

PTC Therapeutics Announces FDA Acknowledgment of New Drug Application Filing for Translarna for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

Eleven Major Patient Groups Outline Joint Position on Health Care Reform

Find MDA in your Community

Find MDA
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