According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
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…
According to a story from m.dailyhunt.in, Vinayak Sreedhar, an Indian student with Duchenne muscular dystrophy, passed away in the midst of taking his exams. Vinayak was a driven and highly…
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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…
Lithium boosts muscle strength in mice with rare muscular dystrophy New drug target identified Click to share on Pinterest (Opens in new window) Removing one gene caused normal muscle muscle…
A press release from American biopharmaceutical company AMO Pharma, published by PR Newswire, detailed the recent completion of the Company's new congenital myotonic dystrophy type 1 (CDM1) symptom-evaluation scale. The…
According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…
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…
According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…
According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization…
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…
Tay-Sachs disease is a rare, genetic and sadly fatal condition. It's a neurodegenerative disorder caused by an impaired production of the β-Hexosaminidase A enzyme (HEXA). Symptoms of this condition include listlessness, diminishing…
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 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…
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…
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…
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