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Muscular dystrophy
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In Depth 4 Apr 2024
Seven diseases CRISPR technology could cure
Here are some of the diseases that scientists aim to tackle using CRISPR technology, testing its possibilities and limits as a medical tool.
In Depth 7 Sep 2023
Hope for patients with life-threatening muscle disorder after years of clinical trial failures
Duchenne muscular dystrophy is a rare condition that is typically treated with the help of steroids, to manage symptoms, as scientists are yet to close in on a cure. However, the first-ever gene therapy for Duchenne muscular dystrophy is set to open doors for further therapeutic research. As we observe World Duchenne Awareness Day on […]
News and Trends 22 Aug 2022
Santhera and ReveraGen announce first patient with Becker muscular dystrophy dosed in FDA-funded pilot study
The first patient has been dosed in a phase 2 pilot study looking at a treatment for muscular dystrophy. Santhera Pharmaceuticals and ReveraGen BioPharma made the announcement that vamorolone is to be assessed in patients with Becker muscular dystrophy (BMD). The study is being funded by the U.S. Food and Drug Administration (FDA). Eric Hoffman, […]
News and Trends 29 Jul 2022
Sarepta looks for quick approval for Duchenne muscular dystrophy gene therapy treatment
U.S. company Sarepta Therapeutics, Inc. said it intends to submit a Biologics License Application (BLA) seeking accelerated approval for SRP-9001 (delandistrogene moxeparvovec) to treat ambulant individuals with Duchenne muscular dystrophy. SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche. “We are delighted to confirm that based on the feedback we […]
News and Trends 7 Jun 2019
CRISPR Therapeutics and Vertex Seal Deal Worth up to €890M
The US giant Vertex Pharmaceuticals is expanding an existing collaboration with the Swiss company CRISPR Therapeutics, paying up to €890M ($1B) to develop treatments for genetic diseases causing muscle weakness using the gene editing tool CRISPR/Cas9. The deal includes €155M ($175M) paid upfront, and potential future payments based on achieving developmental milestones. In exchange, Vertex […]
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News and Trends 28 Jun 2018
Muscular Dystrophy Drug Fails in Phase 2 Trial
The stock of Summit Therapeutics has plunged by 80% after announcing that its drug candidate for Duchenne muscular dystrophy failed to meet the primary and secondary endpoints of a Phase II clinical trial. Duchenne muscular dystrophy is a genetic disease that causes progressive muscle deterioration in children born with the disease. The British-American company Summit […]
News and Trends 27 Feb 2018
This Small Molecule Protects the Muscles of Patients With Duchenne Muscular Dystrophy
Summit Therapeutics’ treatment for the devastating disease, Duchenne muscular dystrophy, reduces inflammation, which could protect muscle fibers from damage. Summit Therapeutics focuses on the development of treatments that can help patients suffering from diseases that currently lack options. The biotech has reported Phase II results for its drug for the genetic disease, Duchenne muscular dystrophy […]
News and Trends 5 Jul 2017
Cutting Through the BS in Biopharma: How To Spot Hype
Faced with the oft-quoted figure that 90% of clinical trials end in failure, how can patients, investors, and readers be spared more disappointment? After discussing failures in biotech at Refresh earlier in June, I headed to Copenhagen last week for the European Conference for Science Journalists to cajole a panel into discussing how some of these stories feature […]
News and Trends 28 Jun 2017
Swiss Scientists Have Designed New Proteins to Treat Muscular Dystrophy
Researchers at the University of Basel have designed two proteins that can reverse congenital muscular dystrophy and increase the lifespan of mice. Congenital muscular dystrophy (CMD) englobes several genetic diseases that cause muscle weakness from birth. As patients age, the disease becomes more severe and affects breathing, often leading to death before adulthood. Unfortunately, there […]
News and Trends 22 Jun 2017
French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy
Noble Généthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease. Dedicated to rare diseases since 1990 and more recently to gene therapy, Généthon is one of very few not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is […]
More News! 30 May 2017
FDA Fast Tracks Muscular Dystrophy Drug backed by Woodford
The FDA has handed out a Fast Track Designation to AMO Pharma for its key asset AMO-02 to treat congenital myotonic dystrophy. The British-American biotech hybrid AMO Pharma is advancing a small pipeline of drugs for rare genetic disorders. The biotech, which is backed by famous UK Investor Neil Woodford, is currently pushing its lead candidate […]
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