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…
A first patient has been dosed in the Phase 2 trial investigating pegcetacoplan (APL-2), Apellis Pharmaceuticals‘ candidate therapy for amyotrophic lateral sclerosis (ALS), the company and the Swedish Orphan Biovitrum (Sobi) announced. The potentially pivotal trial, called MERIDIAN (NCT04579666), is currently enrolling patients…
The ALS Association and I AM ALS have filed a petition with the U.S. Food and Drug Administration (FDA), calling on the regulatory agency and Amylyx Pharmaceuticals to make AMX0035 available to people with amyotrophic lateral sclerosis (ALS) as quickly as possible. Signed by more than 50,000 patients, their caregivers and family, the…
The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Seelos Therapeutics‘ SLS-005 (trehalose), a medication designed to slow the progression of amyotrophic lateral sclerosis (ALS), the company announced in a recent press release. The designation…
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