Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
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Minnesota lawmaker Dave Lislegard has introduced two measures in the state’s legislature that would invest tens of millions of dollars in amyotrophic lateral sclerosis (ALS) research and caregiver support. The first bill, HF 3603, would appropriate $20 million to the University of Minnesota for competitive research grants to…
Note: This story was updated April 4, 2022, to clarify that 700 participants, including both ALS patients and healthy volunteers, have been recruited to the study. Mitsubishi Tanabe Pharma America (MTHA) has joined EverythingALS’s consortium to accelerate the development of new digital biomarkers for amyotrophic lateral…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
Dosing has begun in a Phase 2/3 trial of SLS-005, Seelos Therapeutics’ infusion therapy to slow progression in amyotrophic lateral sclerosis (ALS) that is running as the fifth arm of the multi-regimen HEALEY ALS platform trial. The trial (NCT05136885) plans to…
A company called ProJenX has been launched and will focus on the clinical development of treatments for brain diseases, starting with prosetin, its lead candidate therapy for amyotrophic lateral sclerosis (ALS). ProJenX was created in collaboration with Project ALS, researchers at Columbia University, and Medical Excellence Capital, which…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
The European Medicines Agency (EMA) has agreed to review a marketing authorization application (MAA) from Amylyx Pharmaceuticals requesting the approval of AMX0035 — a combination of two compounds — for the treatment of people with amyotrophic lateral sclerosis (ALS). The validation comes nearly two months after Amylyx filed the…
A research team in Canada received a CA$1.6 million (almost $1.25 million) grant from the Weston Family Foundation to study the benefits of a specific probiotic in people with amyotrophic lateral sclerosis (ALS). Probiotics — found in yogurt, fermented foods, and dietary supplements — are live microorganisms thought to improve…
Health Canada has authorized a new drug submission for masitinib — AB Science’s add-on therapy for amyotrophic lateral sclerosis (ALS) — under the notice of compliance with conditions policy (NOC/c), designed to get new medications to patients more quickly. If granted, this would allow conditional marketing of…
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