According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…
According to a story from Fierce Biotech, the results of a year-long study examining the utility of the groundbreaking gene-editing technology CRISPR in treating the rare genetic disorder Duchenne muscular…
According to a story from BBC, Mats Steen passed away at 25 years old due to complications from his Duchenne muscular dystrophy. Upon his death, parents Robert and Trude grieved…
Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000 male births). It's caused by…
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…
According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001. This investigational product is in…
According to an article published by Washington University of Medicine in St. Louis, scientists appear to have succeeded in slowing axon destruction in mice through standard gene therapy. Though an…
According to a story from bioengineer.org, it has long been known that people with the rare disorder Duchenne muscular dystrophy are at a greater risk of developing a rare cancer…
Being diagnosed with any serious condition can be scary, overwhelming, and full of unknowns. For those who are diagnosed with a rare disease such as Duchenne muscular dystrophy, spinal muscular atrophy,…
According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known…
It has just been announced that Capricor Therapeutics has put their Duchenne Muscular Dystrophy (DMD) clinical trial on hold. Fortunately, the company does plan on continuing the trial. What is…
According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…
According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…
Andrew Taylor celebrated his 50th birthday this year. This is a big year for anyone, but especially for Taylor, who was told he would not make it to the…
This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…
"It's funny to say grief as it is not a bereavement but you kind of are grieving the loss of the child you thought you had and the future they…
Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
According to a story from prnewswire.co.uk, the biopharmaceutical company PTC Therapeutics recently announced the release of initial data from the first drug registry for its product Translarna (ataluren). This registry…
According to a story from Boston Health News, The US Food and Drug Administration's Accelerated Approval program is being used to approve a steadily growing number of drugs in recent…
According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…
The non-profit organisation CureDuchenne has announced that they have made a $500,000 investment in the company 4D Molecular Therapeutics in order to support the development of gene therapy research. For…
According to a recent press release, Pfizer Inc. has terminated two on-going clinical trials that were investigating domagrozumab (PF-06252616) as a potential treatment for Duchenne muscular dystrophy. This decision follows…
The Australian biopharmaceutical company Antisense Therapeutics has announced that the first patient has been dosed on a Phase 2 clinical trial of the investigational drug ATL1102, which is being developed…
Friday September 7th is World Duchenne Muscular Dystrophy Day! Duchenne muscular dystrophy (DMD) is one of nine forms of muscular dystrophy. Patients with DMD cannot make dystrophin in their muscles causing…
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