Muscular dystrophy Archives

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News and Trends 28 Mar 2017

Why did Biotech Beat Pharma to Bring the First Gene Therapy to Market?

Pharma would seemingly best biotech in any competition. A few CEOs explain why it left the honor of “The First Gene Therapy” to biotech. On July 20, 2012, the western world’s first gene therapy, Glybera, received marketing authorization from the EMA. The treatment was designed and developed by uniQure as one-off treatment for Lipoprotein Lipase Deficiency (LPLD), a […]

March 28, 2017 - 5 minutesmins - By Evelyn Warner

crispr therapeutics muscle stem cells anagenesis

News and Trends 10 Jun 2016

CRISPR Thx in yet another Collaboration, this time for Muscle Dystrophy

CRISPR Therapeutics has struck a deal with Anagenesis Biotechnologies, which has key technology for muscle stem cells. Together, they will tackle muscle disorders – including the rare disease Duchenne Muscular Dystrophy (DMD). CRISPR Therapeutics (Basel, Switzerland) is the European champion of gene editing for health applications, a field that promises to revolutionize therapies – even if […]

June 10, 2016 - 3 minutesmins - By Denise Neves Gameiro

News and Trends 1 Dec 2015

Rare Diseases: Still a ‘Blue Ocean’ for Biotechs?

We all know the success stories linked to Rare Disease such as Genzyme. But I wanted to get a deeper understanding on what is today considered as Rare disease, who is acting the field and the reasons for so many Biotech companies to dive into the field. Last week was the RARE 2015 congress in […]

December 1, 2015 - 6 minutesmins - By Philip Hemme

News and Trends 14 Aug 2015

Milestone in Stem Cell Research: European Researchers grow Millimeter-Long Muscle Fibers!

Stem cell research grows and grows in Europe! For the first time ever, researchers succeded to effectively grow a large number of muscle cells derived from pluripotent stem cells! Researchers from six European countries are participating in the PluriMes project to give a cutting edge to the European stem cell research. Launched in February 2014, […]

August 14, 2015 - 2 minutesmins - By Philip Hemme

News and Trends 28 Oct 2014

Stem Cell Therapy for Degenerative Muscle Disease thanks to ENDOSTEM Project

ENDOSTEM project aims to identify an agent capable of ‘boosting’ stem cells already present in muscle tissue for a more efficient tissue repair to tackle degenerative muscle disease. Muscular dystrophy is a type of degenerative muscle disease. One of its most known forms, Duchenne muscular dystrophy, affects 1 over 3 500 boys worldwide. It affects patients progressively, they […]

October 28, 2014 - 3 minutesmins - By Julie Lellouche

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