According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
According to a story from Xtalks, there has been an uptick in research related to rare diseases and the development of new therapies for them. Despite this, only a small…
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…
The Star Gazette recently reported that Alan Ramsay Evans, a 34-year-old man from Horseheads, New York, remains in the hospital after developing respiratory failure. Three decades ago, when he was…
Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…
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…
Duchenne muscular dystrophy (DMD) has gained recent attention. New symptom management and gene therapy treatments are at the forefront of DMD research. DMD is a genetic disease characterized by progressive…
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…
We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…
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…
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