Muscular dystrophy Archives

Best in Biotech 3 Oct 2024

Six biotechs driving progress in Duchenne muscular dystrophy

In this article, we take a look at six clinical-stage biotechs that are fostering R&D in the Duchenne muscular dystrophy therapeutic space.

October 3, 2024 - 8 minutesmins - By Roohi Mariam Peter

In Depth 3 May 2024

Can emerging new treatments lead the way to cure Duchenne muscular dystrophy?

Delve into the new treatment approaches to Duchenne muscular dystrophy, a challenging disease for those suffering from it, and research.

May 3, 2024

Best in Biotech 16 Apr 2024

Seven biotech companies climbing their way up in Colorado

We take a look at seven biotechs in Colorado that have secured funding in recent years, to make headway with their pipeline candidates.

April 16, 2024 - 8 minutesmins - By Roohi Mariam Peter

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.

April 4, 2024 - 11 minutesmins - By Clara Rodríguez Fernández

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, […]

August 22, 2022 - 3 minutesmins - By Liza Laws

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 […]

July 29, 2022 - 3 minutesmins - By Jim Cornall

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 […]

June 7, 2019 - 2 minutesmins - By Jonathan Smith

Duchenne muscular dystrophy Summit Therapeutics

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 […]

June 28, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

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 […]

February 27, 2018 - 3 minutesmins - By Alex Dale

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 […]

July 5, 2017 - 4 minutesmins - By Evelyn Warner

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 […]

June 28, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

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 […]

June 22, 2017 - 3 minutesmins - By Evelyn Warner

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Muscular dystrophy

Six biotechs driving progress in Duchenne muscular dystrophy

Can emerging new treatments lead the way to cure Duchenne muscular dystrophy?

Seven biotech companies climbing their way up in Colorado

Seven diseases CRISPR technology could cure

Santhera and ReveraGen announce first patient with Becker muscular dystrophy dosed in FDA-funded pilot study

Sarepta looks for quick approval for Duchenne muscular dystrophy gene therapy treatment

CRISPR Therapeutics and Vertex Seal Deal Worth up to €890M

Muscular Dystrophy Drug Fails in Phase 2 Trial

This Small Molecule Protects the Muscles of Patients With Duchenne Muscular Dystrophy

Cutting Through the BS in Biopharma: How To Spot Hype

Swiss Scientists Have Designed New Proteins to Treat Muscular Dystrophy

French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy

Subscribe to our newsletter to get the latest biotech news!

10 biotech companies to watch in 2025

Biotech in 2024: A retrospective

Grab your free report on 3D organoids and explore their future potential

Five biosimilar companies you should know about

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

10 biotech companies to watch in 2025

Biotech in 2024: A retrospective

Grab your free report on 3D organoids and explore their future potential

Five biosimilar companies you should know about

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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