WILMINGTON, Del. — AstraZeneca Pharmaceuticals LP and Ionis’ WAINUA™ (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered...
treatment News
CAMBRIDGE, Mass. — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced it has regained full rights to WVE-006, an investigational GalNAc-conjugated RNA editing oligonucleotide (AIMer) for alpha-1 antitrypsin deficiency (AATD), from GSK. This...
CAMBRIDGE, Mass. — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive data from the 200 mg single and multidose and 400 mg single dose cohorts of the ongoing Phase 1b/2a RestorAATion-2 study...
CAMBRIDGE, Mass. — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive data from the Phase 2 FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne...
WASHINGTON DC – Gene-editing technologies are progressing fast, and there are now more than 100 ongoing trials for new therapies and diagnostics based on CRISPR-Cas, zinc finger nucleases, TALENS, and base editors. While most of these trials are evaluating ex vivo gene-edited cell-based therapies for cancers and genetic blood disorders,...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced topline results from Week 52 of COACH, the first Phase 2 clinical trial to evaluate combination therapy with once-weekly TransCon CNP (navepegritide) and once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia. At Week 52, combination therapy showed durable growth without...
Guildford, England. Insulin icodec, a once-weekly basal injection to treat type 1 diabetes, has the potential to be as effective in managing the condition as daily basal insulin treatments, according to research from the University of Surrey. The results of the year-long phase 3 clinical trial could revolutionise the future...
MISSISSAUGA, Ontario – Novo Nordisk announced today that Health Canada has issued marketing authorization with conditions [Notice of Compliance with Conditions (NOC/c)] for Wegovy® (semaglutide injection) for the treatment of non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) in adults with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis).1 Authorization in Canada...
PLAINSBORO, N.J. — Novo Nordisk today announced that the U.S. Food and Drug Administration (FDA) has approved a new indication for Wegovy® (semaglutide) injection 2.4 mg, making it the first and only glucagon-like peptide-1 receptor agonist (GLP-1 RA) FDA-approved to treat adults with MASH with moderate to advanced liver scarring (fibrosis), but...
New York, NY – Patients who have drug-resistant tuberculosis (TB) have a similar microbiological response to bedaquiline-based second-line medications as patients with drug-sensitive TB taking first-line regimens, according to researchers at Weill Cornell Medicine in New York and GHESKIO Centers in Haiti. Second-line medications are those that are given when...