#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

Researchers have discovered that C9orf72 — the gene whose mutated version is associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) — works by activating key proteins for the transport and degradation of molecules inside the cell, in normal conditions. This discovery expands knowledge on how C9orf72 may contribute to the…

A research team led by Northwestern University has been granted $12.6 million by the National Institute on Aging at the National Institutes of Health (NIH) to study the role of protein quality control in aging and neurodegenerative diseases such as Alzheimer’s…

Renowned physicist and author Stephen Hawking, perhaps the most famous ALS sufferer after Lou Gehrig, had his final book posthumously published on Oct. 16. In “Brief Answers to the Big Questions,” one of the answers Hawking offers is that there is…

The University California San Diego (UC San Diego) has licensed the right to develop and commercialize an investigational gene therapy for amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, called SynCav1, to CavoGene LifeSciences. The therapy may also benefit people with traumatic brain and spinal cord injuries, and cognitive decline disorders.

ALS life involves a lot of waiting. We wait for people to help us with daily tasks, we wait for medical test results, and we wait for new ALS symptoms. But it’s the constant thoughts of despair and hopelessness that put us in what I call the ALS…

Despite a lack of clinical evidence, complementary and alternative medicines (CAM) are often used by patients with amyotrophic lateral sclerosis (ALS), sometimes in combination with conventional medications, according to a review study. With scarce evidence on the safety and effectiveness of CAM in ALS and the possible risks they…

RNA molecules that are associated with genetic disorders characterized by damage to nerve fibers are found at higher-than-usual numbers in compartments of nerve cells, a new study reports. This finding may lead to new ways of treating neuromuscular diseases such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy and…

Swiss biopharma GeNeuro has secured the exclusive worldwide license to the clinical development program for a pHERV-K Env antibody after preclinical results showed its potential to treat amyotrophic lateral sclerosis (ALS). The company exercised its option for the rights to the investigational therapy under the terms of a January 2017…

Voyager Therapeutics’ experimental therapy VY-SOD102 shows promise for treating patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results in animal models. The preclinical data were presented at the Congress of the European Society of Gene and Cell Therapy…