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May 21, 2018May 21, 2018

Protein That Helps to Regulate Innate Immune System Seen to Slow ALS Progression in Early Study in Mice

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The protein TRIF can prevent resident immune cells of the brain from activating abnormally and slow the progression of amyotrophic lateral sclerosis (ALS), researchers in Japan working in a mouse model ... Read more

May 17, 2018May 17, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

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The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

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The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 17, 2018May 17, 2018

Specially Engineered Neural Cells Delay Disease and Extend Life in ALS Animal Model

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Transplanting engineered neural cells into the brain of an amyotrophic lateral sclerosis (ALS) animal model delayed disease progression and extended the animals’ survival, a study shows. The study, “Transplantation of Neural ... Read more

May 16, 2018May 16, 2018

MDA Grants Include Research Projects for ALS and Other Diseases

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The Muscular Dsytrophy Association (MDA) awarded 13 new grants totaling $2.6 million to find treatments for neuromuscular disorders, including amyotrophic lateral sclerosis (ALS). The 13 grants are divided into three ... Read more

May 15, 2018May 15, 2018

Processes Driving FUS Proteins to Clump in ALS and Common Dementia Described in Study

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The distinct processes that drive FUS proteins to aggregate and clump in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) were detailed in a new report. The study, “Phase Separation ... Read more

May 14, 2018May 14, 2018

Worldwide Partners with Datavant to Improve Clinical Trials in ALS, Other Diseases

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Worldwide Clinical Trials and the healthcare technology company Datavant are partnering in an effort to improve trials in neurological diseases such as amyotrophic lateral sclerosis (ALS). With the aging of ... Read more

May 11, 2018May 11, 2018

MIT Researchers Find Potential Role of C9orf72 Gene in ALS, Frontotemporal Dementia

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Mutations in the C9orf72 gene deregulate the process by which cells remove waste, leading to a buildup of toxic elements inside them, researchers have found. This discovery expands knowledge ... Read more

May 10, 2018May 10, 2018

Biohaven Establishes Expanded Access Program for Investigational ALS Therapy BHV-0223

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Biohaven Pharmaceuticals has established an expanded access program (EAP) for patients with amyotrophic lateral sclerosis (ALS) for its investigational treatment candidate, BHV-0223, the company recently announced. The United States Food ... Read more

May 9, 2018May 9, 2018

Alnylam Testing New Molecular Therapy Candidates for ALS, Other Neurodegenerative Diseases

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Newly developed molecular treatment candidates called small interfering RNAs (siRNAs) have been successfully delivered to the brain and spinal cord in a rat preclinical study, Alnylam Pharmaceuticals recently announced. According ... Read more

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