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…
According to a story from the Yale Daily News, Brian Wallach, class of 2003, was diagnosed with amyotrophic lateral sclerosis, or Lou Gehrig's disease, in November of 2017. Although being…
According to a story from EurekAlert!, a team of scientists has discovered a genetic variation that can cause amyotrophic lateral sclerosis. This variant acts on a biologic pathway that has…
Researchers at BGN Technologies (at Ben-Gurion University) have come up with a new way to predict and monitor progression of a wide array of neurodegenerative diseases. One of the illnesses…
The Importance of Speech The ability to speak gives individuals not only the opportunity to communicate with one another, it provides an outlet for expression, creativity, and connectedness. Unfortunately, that…
According to a story from pm360online.com, the Orion Corporation, a pharmaceutical company based in Finland, has recently announced that it plans to continue its Phase 3 trial of levosimendan as…
According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…
Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…
According to a story from brightsurf.com, a team from the Washington University School of Medicine, based in St. Louis, has succesfully developed a gene therapy that appears to prevent damage…
The Florey Institute of Neuroscience (FIN), one of the world’s largest and most respected brain research centers, recently announced trial results for the drug CuATSM, developed by FIN, the School…
According to an article published by R&D, scientists at Harvard have identified a new genetic clue that they suspect might be linked to the development of amyotrophic lateral sclerosis (ALS).…
Perhaps the most difficult thing about rare disease research is that not only do the conditions affect only a small portion of the population, but like every diagnosis, they affect…
According to a story from king5.com, nine-year-old Taylor Futch's smile has always been able to captivate those around her. Unfortunately, Taylor's smile is often the extent of her ability to…
According to a story from the USC News, a team of doctors with the University of Southern California (USC) ALS Clinic are doing their best to help Shaun Kalpakoff, a…
Amyotrophic Lateral Sclerosis (ALS) stops communication between the brain and voluntary muscles. Patients slowly lose muscle strength, affecting their ability to walk, talk, and even breathe. The illness eventually results…
Neurodegenerative disorders affect millions of people across the world. They include Amyotrophic Lateral Sclerosis, Parkinson's disease, Huntington's disease, and Alzheimer's disease, among others. For most neurodegenerative diagnoses, there are no effective treatments, no…
According to a story from the Centers for Disease Control and Prevention, the National ALS Registry is playing a vital role in learning more about amyotrophic lateral sclerosis, a devastating…
Amyotrophic lateral sclerosis (ALS) is a condition which affects the bodies nerve cells. It's neurodegenerative and progressive, causing the patient muscle weakness and muscle atrophy. It eventually results in paralysis.…
According to a story from Buzzfeed News, Stephen Hillenburg, the creator of the smash hit children's cartoon SpongeBob SquarePants has passed away at age 57. Last year, Hillenburg had revealed…
In 1993, researchers found one of the causes of familial amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder which affects the motor neurons. Motor neurons are responsible for transmitting…
According to a story from telegram.com, a group of researchers from the University of Massachusetts medical school recently announced that they may have reached a major treatment breakthrough for genetically…
According to a recent article by UMass Medical School, promising new research from scientists at UMass supports the potential of gene therapy as a treatment for amyotrophic lateral sclerosis. The…
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.…
According to a story from Newswire, the specialty pharmaceutical company Tikomed recently announced that the first patient has been dosed in an clinical trial testing the company's experimental product ILB.…
According to a story from The Examiner, Sarah Nauser-Olsen was at a great place in her life: she had a stable job as a police officer and was also well…
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