Clinical Trial Initiated for New Technology That Could Allow Amyotrophic Lateral Sclerosis Patients to Communicate

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…

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Could a “Gene Expression Atlas” Unlock the Secrets of Amyotrophic Lateral Sclerosis?

According to a story from MD Magazine, the team at Spatial Transcriptomics has utilized an innovative computational approach in order to map gene expression on the spinal cords of patients…

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The Struggles of being a Rare Disease Patient in China

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…

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After Diagnosis, a Yale Alum Establishes Nonprofit Focused on Amyotrophic Lateral Sclerosis
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After Diagnosis, a Yale Alum Establishes Nonprofit Focused on Amyotrophic Lateral Sclerosis

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…

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Can Amyotrophic Lateral Sclerosis Patients Speak Again With New Technology?

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…

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Company Plans Extension Study for Phase 3 Trial of Amyotrophic Lateral Sclerosis Drug
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Company Plans Extension Study for Phase 3 Trial of Amyotrophic Lateral Sclerosis Drug

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…

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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial
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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial

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…

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Results from Phase II/III Trial Bring Hope for FDA Approval for New Niemann-Pick Disease Type C Treatment

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…

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Nerve Damage Blocking Gene Therapy Could be Useful in a Variety of Rare Diseases
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Nerve Damage Blocking Gene Therapy Could be Useful in a Variety of Rare Diseases

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…

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National Geographic Features Innovative Research That Will Advance Precision Medicine

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…

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Girl With Extremely Rare Juvenile Amyotrophic Lateral Sclerosis Keeps Positive Attitude
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Girl With Extremely Rare Juvenile Amyotrophic Lateral Sclerosis Keeps Positive Attitude

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…

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A Team From The University of Southern California Take on Single Father’s Amyotrophic Lateral Sclerosis
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A Team From The University of Southern California Take on Single Father’s Amyotrophic Lateral Sclerosis

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…

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CZI has Committed 64 Million Dollars to the Research of Neurodegenerative Disorders

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…

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The Vital Role of the National Amyotrophic Lateral Sclerosis (ALS) Registry
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The Vital Role of the National Amyotrophic Lateral Sclerosis (ALS) Registry

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…

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Potential New Treatment for Amyotrophic Lateral Sclerosis which Doesn’t Require Swallowing!

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.…

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