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Bioscience Companies in Ohio Take Aim at Rare Diseases
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…
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The Barriers to Developing Rare Disease Drugs
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…
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FDA Releases New Info About How it Could Regulate Gene Therapy
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…
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CRISPR Successfully Treats Duchenne Muscular Dystrophy in Mouse Model
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…
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Family Touched By Muscular Dystrophy Sets Up GoFundMe
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…
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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects
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…
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Online Gaming Allowed Duchenne Muscular Dystrophy Patient to Transcend Physical Limits
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…
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New Funding Program to Support Newborn Screening for Duchenne Muscular Dystrophy
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…
HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy is on Track to Resume
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…
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Company Releases Early Data and Plans to Boost Dose in Duchenne Muscular Dystrophy Trial
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…
Curing DMD: Collaborating All Aspects of Care
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…
Gene Therapy Halts Axon Destruction in Mice
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…
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New Research Highlights the Link Between Duchenne Muscular Dystrophy and Rhabdomyosarcoma
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…
Australian Government Initiates New Trial Program to Support Individuals Recently Diagnosed with a Rare Disease
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,…
<a href="https://pixabay.com/users/Aymanejed/">Aymanejed</a> / Pixabay
Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial
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…
Clinical Trial for Duchenne Muscular Dystrophy now on Hold
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…
<a href="https://pixabay.com/users/931527/">931527</a> / Pixabay
ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial
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…
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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients
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…
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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child
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…
The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy
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…
Grief After Diagnosis: Rare Families Need a Specialized Form of Support
"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…
This Just In: India Never Implemented their Rare Disease Policy as Promised
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…
A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!
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…
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Participants in a Duchenne Muscular Dystrophy Clinical Trial Found out the Drug Wasn’t Working on Social Media
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…
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Patient Registry Demonstrates The Effectiveness of Translarna in Duchenne Muscular Dystrophy
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…
