H.R. 2408 H.R. 2408 is a new piece of federal legislation otherwise known as the "Ensuring Access to Quality Complex Rehabilitation Technology Act." Essentially, this act creates a new category…
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The News Hub at Washington State University recently carried an article describing initial success by researchers towards developing a drug for a rare form of muscular dystrophy. Several thousand people…
According to a press release from Massachusetts-based biotech company Fulcrum Therapeutics, the company has successfully secured global commercialization rights of GlaxoSmithKline-developed experimental facioscapulohumeral muscular dystrophy (FSHD) drug losmapimod. Losmapimod, an…
A recent publication from Charcot-Marie-Tooth News announced that this year, for the first time ever, the Muscular Dystrophy Association will be hosting its annual clinical conferences at the same event…
People living with rare diseases such as amyotrophic lateral sclerosis (ALS) or muscular dystrophy as well as those who have suffered from a stroke or spinal cord injury often experience…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
A recent study published in Cell has exhibited the scientific community's negligence for conducting research that includes an adequate representation of non-Europeans. Specifically, it showed that 78% of patients in genomic…
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…
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…
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…
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…
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…
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…
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…
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…
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