News

Actimed Therapeutics has obtained the rights to develop and commercialize ACM-002 (S-oxprenolol) for the treatment of amyotrophic lateral sclerosis (ALS) in Europe, the company announced. The rights, licensed from patent owner Charité University Medicine Berlin, cover all major European markets, including France, Germany, the U.K., Italy,…

The U.S. Senate has passed a bill that may make it easier for people with amyotrophic lateral sclerosis (ALS) to access disability benefits. The proposed legislation, the ALS Disability Insurance Access Act of 2019, still must be approved by the U.S. House of Representatives. If enacted, the legislation…

The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention (CDC) to recommend that people living with neuromuscular diseases (NMDs) have early access to any federally approved COVID-19 vaccine. The MDA made its request in a letter to members of the…

Canadian authorities have approved Xeomin (incobotulinumtoxinA) for the treatment of chronic sialorrhea, or excessive drooling, associated with neurological diseases such as amyotrophic lateral sclerosis (ALS). The decision makes Xeomin the first and only neurotoxin for chronic sialorrhea licensed for this indication in Canada. The therapy was…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Two amyotrophic lateral sclerosis (ALS) staging systems — the King’s and the Milano-Torino’s, known as MiToS — may effectively assess changes in patients’ clinical progression and treatment responses, a study suggests. The data also supported the previously reported complementary nature of these two systems, showing that the…

New research into the motor networks of polio survivors shows the brain may “rewire” itself in neurological diseases, leading to a reorganization that also may serve as a potential biomarker of amyotrophic lateral sclerosis (ALS) and other motor neuron disorders, a study reports.  Published in…

A first set of Canadian guidelines for the care of people with  amyotrophic lateral sclerosis (ALS) have been published, emphasizing the need for a holistic and patient-centered approach, with attention to emotional well-being. The guidelines, reported to include comprehensive and easy-to-reference best practice…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

BREN-02, a lab-made form of the human protein engrailed 1 (EN1), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) as a potential treatment of amyotrophic lateral sclerosis…