It has only been a month since AstraZeneca’s $39 billion takeover of Alexion was completed, but the merger has already had a setback. Alexion has dropped development of its Ultomiris drug in amyotrophic lateral sclerosis (ALS), chalking up the first clinical failure for the drug since AZ took control. The...
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Leiden, Netherlands– Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW study. The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the...
LEIDEN, Netherlands — Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that the first patient has been dosed in the company’s pivotal, multicenter Phase 3 clinical program to evaluate the safety and efficacy of the company’s lead asset,...
LEIDEN, the Netherlands — Azafaros, a company aiming to become a leader in lysosomal storage disorders (LSDs), focused on addressing especially neurological symptoms, today announced the publication of proof-of-concept preclinical data with its lead product, nizubaglustat, in GM2 gangliosidosis. The data, published in the 7 January issue of the Journal of...
Patients with idiopathic pulmonary fibrosis (IPF) being treated with low dose azithromycin for cough did not see significant improvements compared with placebo, according to the results of a recent study published in the Annals of the American Thoracic Society. Researchers conducted a double-blind, randomized placebo-controlled cross-over clinical trial (ClinicalTrials.gov Identifier: NCT02173145) treating...
AzurRx BioPharma Announces Completion of Enrollment in First Cohort of Phase 2b OPTION 2 Extension Study of MS1819 DELRAY BEACH, Fla., Jan. 28, 2021 – AzurRx BioPharma, Inc. (“AzurRx” or the “Company”) (NASDAQ: AZRX), a company specializing in the development of targeted non-systemic, recombinant therapies for gastrointestinal (GI) diseases, today...
DELRAY BEACH, Fla. – AzurRx BioPharma, Inc. (NASDAQ: AZRX), (“AzurRx” or the “Company”), a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced positive interim data from the first 18 out of 20 patients in its Phase 2 trial evaluating MS1819...
Skelly’s Bar in Ballymahon is to play host to a special fundraising night in aid of the Friedeichs Ataxia Society of Ireland on Friday, June 13 next with a host of excellent local acts performing on the occasion. Friedeichs Ataxia is a rare genetic and progressive disorder of the central...
WASHINGTON, D.C. — Muscular dystrophy is one of the most common genetic disorders. One in every 3,500 baby boys is diagnosed with the condition. The most devastating type — Duchenne muscular dystrophy — slowly robs kids of their muscles, and they rarely live past age 20. Researchers say they’ve made...
Tel Aviv, Israel – Autism Spectrum Disorder (ASD), a neurodevelopmental condition characterized by decreased social communication and repetitive behaviors, has long intrigued scientists seeking to unravel its underlying mechanisms. Researchers from the Azrieli Faculty of Medicine of Bar-Ilan University have now identified a potential link between ASD and the composition...