The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
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…
Time for Revision Like many things in this world, research for rare diseases such as Spinal Muscular Atrophy (SMA), Multiple Sclerosis, and Duchenne Muscular Dystrophy (DMD) has evolved substantially in recent years. This evolution…
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…
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…
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…
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…
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…
"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…
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…
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…
"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…
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…
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.…
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…
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…
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…
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…
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.…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
