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September 24, 2018September 24, 2018

Misfolded SOD1 Protein May Have Key Role in Sporadic ALS, Study Suggests

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Specific patterns of a misfolded protein — superoxide dismutase 1 (SOD1) — are found in the brain and spinal cord of patients with sporadic amyotrophic lateral sclerosis (ALS), according to ... Read more

September 24, 2018September 24, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products ... Read more

September 21, 2018September 21, 2018

Parkinson’s Therapy Azilect Added to Rilutek May Delay Fast-progressing ALS, Phase 2 Trial Shows

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Adding Azilect (rasagiline) — a medication already approved to treat Parkinson’s disease — to Rilutek (riluzole) may be able to slow the advancement of fast-progressing amyotrophic lateral sclerosis (ALS), a Phase ... Read more

September 20, 2018September 20, 2018

ALS, Frontotemporal Dementia Have Distinct Patterns of Brain Inflammation, Study Shows

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Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are caused by different patterns of inflammation in the brain, which may help explain why these two illnesses are so distinct. ... Read more

September 19, 2018September 19, 2018

New Method Described in Thesis Can Help Characterize and Target Misfolded Protein in ALS

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A newly developed method can help characterize the types of misfolded proteins present in patients with amyotrophic lateral sclerosis (ALS), according to findings in a thesis. The recently published ... Read more

September 18, 2018September 18, 2018

Retrotope’s Experimental RT001 Now Available for ALS Under Expanded Access Program

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Retrotope is providing its investigational fatty acid being developed for Friedreich’s ataxia, RT001, to people with amyotrophic lateral sclerosis (ALS) under an expanded access program, also called “compassionate use.” Individual ... Read more

September 17, 2018September 17, 2018

New Method to Create Human Neural Stem Cells Can Regenerate Spine in Rats, Study Shows

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Human spinal cord neural stem cells (NSC), created using an innovative method, were seen to regenerate functional neurons in the damaged tissue of rats with spinal injuries, according to ... Read more

September 14, 2018September 14, 2018

Problems with Behavior and Thinking Skills Evident in ALS Patients and Progress with Disease, UK Study Says

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Amyotrophic lateral sclerosis’ toll on the thinking abilities and behavior of patients is as progressive and damaging as the disease’s physical toll, researchers in the U.K. report, with ALS-specific cognitive ... Read more

September 13, 2018September 13, 2018

Protein Staufen1 Could Be Target for Future ALS Therapies, Mouse Study Suggests

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Depleting a protein called Staufen1 improved motor function and lowered protein aggregation in a mouse model of spinocerebellar ataxia type 2 (SCA2). The findings suggest that targeting Staufen1 could ... Read more

September 12, 2018September 12, 2018

Challenges, Benefits of Noninvasive Mechanical Ventilation for ALS Patients Reviewed in Study

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Survival and quality of life of patients with more advanced amyotrophic lateral sclerosis (ALS) can be improved significantly with the use of noninvasive mechanical ventilation (NIV). But many factors can ... Read more

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