SAN DIEGO, Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia’s submission. “Rett syndrome is a profoundly debilitating and complex...
treatment News
SAN DIEGO. Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the journal Developmental Medicine and Child Neurology published interim results from the caregiver-reported observational online, open-label, ongoing LOTUS study evaluating effectiveness and tolerability outcomes in patients with Rett syndrome who are prescribed DAYBUE® (trofinetide) under routine clinical care in the U.S. Findings included reported...
According to the results of a phase 1/2 study, the Bruton tyrosine kinase (BTK) inhibitor acalabrutinib was safe and efficacious, with durable remissions, in patients with previously untreated chronic lymphocytic leukemia (CLL). The findings were reported in Blood. The single-arm study, ACE-CL-001 (ClinicalTrials.gov Identifier: NCT02029443), evaluated efficacy and safety of acalabrutinib monotherapy...
Acasti Pharma Inc., a late-stage, specialty pharma company advancing three clinical stage drug candidates addressing rare and orphan diseases, recognizes and celebrates Rare Disease Day (February 28), as established by the European Organization for Rare Diseases.
CDER’s Accelerating Rare disease Cures (ARC) Program harnesses CDER’s collective expertise and activities to provide strategic overview and coordination of CDER’s rare disease activities. CDER’s ARC Program is governed by leadership from across CDER’s Office of the Center Director, Office of New Drugs, and the Office of Translational Sciences. The program...
Intas and its subsidiary Accord Healthcare lead commercialisation in Europe and India. In December 2022, serplulimab received orphan drug designation from the EC for the treatment of SCLC, this was recently reviewed by the committee and renewed. ESMO has scored serplulimab 4 out of 5 on their magnitude of clinical benefit scale (MCBS) in...
St. Louis, MO – A drug used to treat children with epilepsy prevents brain tumor formation and growth in two mouse models of neurofibromatosis type 1 (NF1), according to a study by researchers at Washington University School of Medicine in St. Louis. NF1 is a genetic condition that causes tumors...
Heidelberg, Germany – A common over-the-counter ingredient in many cough syrups may have a greater purpose for people suffering from lung fibrosis that is related to any number of serious health conditions. Scientists from EMBL Heidelberg were part of a collaborative effort to discover an effective treatment for lung fibrosis...
London, UK – A single test to speed up diagnosis of a serious disease in pregnant women does not need to be repeated, new research has found. Results from the PARROT-2 trial, published today in the Lancet by researchers from King’s College London and funded by Jon Moulton Charitable Trust,...
Chicago, Illinois – A new antibiotic that works by disrupting two different cellular targets would make it 100 million times more difficult for bacteria to evolve resistance, according to new research from the University of Illinois Chicago. For a new paper in Nature Chemical Biology, researchers probed how a class of synthetic...