Boy with Muscular Dystrophy Speaks to Politicians About Rare Disease
Cameron Dixon is a 17-year-old boy living with muscular dystrophy. His family was first told that he would most likely never be able to walk, eat, or talk, and that…
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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…
Potential Gene Therapy for Limb Girdle Muscular Dystrophy Type 2D Granted Orphan Drug Designation
Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…
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…
Building Community with Charcot-Marie-Tooth Disease
"It helps to share experiences with others who understand what I am going through." Jill Price, a mom with Charcot-Marie-Tooth Disease talks about how social media has helped create a…
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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…
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34 Years After Bhopal Disaster, Chronic Health Conditions Persist
Earlier this month marked a solemn 34 years since the horrendous Bhopal disaster. The worst industrial disaster in human history killed over 3,800 almost instantly, and tens of thousands in…
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…
Study Shows Importance of Biobanks For Rare Disease Research
Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…
Researchers were Wrong about the Cause of Muscular Dystrophy
For a long time, neuromuscular diseases such as muscular dystrophy, ALS, and inclusion body myopathy were thought to be caused as a result of toxic clumps of proteins such as TDP-43.…
Collaboration Improves Outcomes for Muscular Dystrophy Patients
Any research that's done with rare disease patients in mind, working to improve their treatment and experience is wonderful. However, when collaboration between both researchers and organizations working to support…
Pfizer Drops Duchenne Muscular Dystrophy Treatment
According to a report by biopharmadive.com, Pfizer announced they will be dropping support for an experimental Duchenne muscular dystrophy treatment. The company intends to see if the treatment may be…
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A Greater Number of Drugs Are Getting Cleared With the FDA’s Accelerated Approval. What Does This Mean?
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…
Neuromuscular Disease Research Center Opens in Ottawa
A new neuromuscular research center has opened at the Ottawa Hospital. The new center will allow patients living with neuromuscular conditions, such as muscular dystrophy and ALS, to be a…
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Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted
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.…
Muscular Dystrophy Spun Her Around. Now She’s Bringing People Together
Susan Aumiller’s story begins with a dream. She and her husband, Bob, made it a goal to visit every national park in the United States. About halfway through the journey,…
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Cryo-Electron Imaging Provides New Info About Virus Vectors in Gene Therapy
According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
Ribitol Could Benefit Patients With Rare Limb Girdle Muscular Dystrophy and Related Diseases
A recent study published in the journal Nature Communications highlights a potential therapy for the treatment of muscular dytrophies and other diseases linked to mutations of the FKRP gene. In the study, the…
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Experimental Duchenne Muscular Dystrophy Drug Gets Multiple Designations From the FDA
According to a story from Globe Newswire, the biotechnology company Wave Life Sciences recently announced that its investigational product WVE-210201 has received both Rare Pediatric disease designation and Orphan Drug…
This SLP’s Mission to Expand Resources for Those with ALS, Muscular Dystrophy, and More
According to ASHA.org, when Tiffany Turner graduated, she set out to expand opportunities for fellow speech language pathologists like herself. Turner saw a great need in her community for…
Technological Breakthrough for DMD Research
According to News Medical, a team of researchers from London have created new technologies that may aid in further understanding Duchenne muscular dystrophy (DMD). Not only this, but the new…
