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Companies Related to "Sarepta Genethon collaborate Duchenne muscular dystrophy" [Most Relevant Company Matches] RSS

15:50 EDT 25th June 2018 | BioPortfolio

Here are the most relevant search results for "Sarepta Genethon collaborate Duchenne muscular dystrophy" found in our extensive corporate database of over 50,000 company records.

Showing "Sarepta Genethon collaborate Duchenne muscular dystrophy" Companies 1–25 of 195

Extremely Relevant

Sarepta Therapeutics, Inc.

Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world's most ...


Little Hercules Foundation and Team Joseph

Little Hercules Foundation is a registered, non-profit 501(c)(3) organization based in Dublin, Ohio. Little Hercules Foundation got its start in January 2013 when three moms, two of whom had sons diagnosed with Duchenne Muscular Dystrophy, decided to host events to help fund research. Since then, Little Hercules Foundation has grown into much more. We focu...

CureDuchenne Ventures LLC

CureDuchenne Ventures LLC collaborates with pharmaceutical and biotechnology companies to facilitate the development of drugs to treat Duchenne muscular dystrophy. CureDuchenne Ventures LLC was formed by CureDuchenne, a national nonprofit that has funded seven research projects that have advanced to human clinical trials. CureDuchenne has leveraged $100 mi...


Sarepta Therapeutics

Innovative RNA-Based Technology; First-in-Class Therapeutics Sarepta Therapeutics – formerly AVI BioPharma – remains focused on developing first-in-class, RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. Sarepta’s unique technology has yielded a diverse pipeline of RNA-based therapeutics that...

Prosensa Holding N.V.

Prosensa is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington’s disease.

Santhera Pharmaceuticals Holding Ltd

Santhera Pharmaceuticals is a Swiss biopharmaceutical company focusing on the discovery, development and marketing of small molecule pharmaceutical products for the treatment of neuromuscular diseases. Santhera’s vision is to become the leading specialty pharmaceuticals company offering therapies for a number of indications in this area of high unmet medical need which includes many orphan indic...

Retrophin, LLC

Retrophin, LLC is a privately-held New York-based, biotechnology company focused on discovering and developing treatments for rare and life-threatening diseases. Retrophin is currently developing treatments for focal segmental glomerulosclerosis, Duchenne Muscular Dystrophy, spinal muscular atrophy, cystic fibrosis and myotubular myopathy. Retrophin's Seri...

Jett Foundation

Based in Kingston, MA the Jett Foundation is a non-profit organization dedicated to increasing worldwide awareness of Duchenne Muscular Dystrophy with the purpose of raising and appropriating funds for research to realize a cure. After almost 10 years of steady progress in the fight against this dreadful disease, the Jett Foundation has funded promising re...

Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare ...

Akashi Therapeutics, Inc.

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem i...

Akashi Therapeutics Inc.

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare ...

Summit Therapeutics plc

Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease Clostridium difficile infection.

CureDuchenne

CureDuchenne is a nonprofit organization that raises awareness and funds specifically aimed at taking on Duchenne Muscular Dystrophy (DMD). By working closely with the world’s leading DMD scientists CureDuchenne works to determine the most viable research projects that will accelerate the clinical trial process and bring potential life saving drugs to...

Solid GT

Solid GT, a subsidiary of Solid Biosciences, is a gene therapy platform focused on developing novel, disease-modifying therapeutics for Duchenne muscular dystrophy. The company’s licensed technology has demonstrated efficacy in various DMD animal models and lead candidates are currently undergoing IND-enabling studies. Founded in 2014, Solid GT is col...

Relevant

Summit Corporation plc

Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease Clostridium difficile infection. Summi...

Muscular Dystrophy Association

Muscular Dystrophy Association (MDA)

The Muscular Dystrophy Association

Retrophin, Inc.

Retrophin is a biotechnology company focused on discovering and developing treatments for rare and life-threatening diseases. Retrophin is currently developing treatments for focal segmental glomerulosclerosis (FSGS), Pantothenate Kinase-Associated Neurodegeneration (PKAN), Duchenne Muscular Dystrophy and other catastrophic diseases. The company’s lea...

Parent Project Muscular Dystrophy

Genethon and AFM (French Muscular Dystrophy Association)

Muscular Dystrophy Association and Vantage Mobility International

Tivorsan Pharmaceuticals, Inc.

Tivorsan Pharmaceuticals, Inc. is a protein therapeutics company pioneering a unique approach to treating serious neuromuscular disorders, including DMD and Becker Muscular Dystrophy (BMD). This method, using recombinant human biglycan, is based on 25 years of basic science work in the Fallon laboratory at Brown University, Providence, RI. Tivorsan was for...

Biotechnology Industry Organization (BIO) and Parent Project Muscular Dystrophy (PPMD)

Amsterdam Molecular Therapeutics B.V

AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. This safe and efficacious proprietary platform offers a unique manuf...


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