ACHONDROPLASIA

Drug may reduce SIDS in children with achondroplasia

via Murdoch Children’s Research Institute A drug that boosts bone growth in children with achondroplasia may also reduce their chances of sudden infant death syndrome (SIDS), sleep apnoea and needing…

Treatment · Vosoritide

Vosoritide therapy in children with achondroplasia aged 3−59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial

Source: The Lancet Child & Adolescent Health Authors: Prof Ravi Savarirayan, MD • Prof William R Wilcox, MD • Prof Paul Harmatz, MD • John Phillips III, MD • Lynda…

Ascendis · TransCon CNP

Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective in ACcomplisH China Phase 2 Trial

SOURCE: VISEN Pharmaceuticals (Nov 16th, 2023, Shanghai, China) – VISEN Pharmaceuticals (VISEN), an innovative biopharmaceutical company focused on endocrine diseases, is pleased to announce the topline results from the ACcomplisH…

Achondroplasia · International · Vosoritide

Health Ministry announces introduction of Achondroplasia drug for children patients

SOURCE: AGENDA.GE Agenda.ge, 14 Nov 2023 – 15:18, Tbilisi,Georgia Vosoritide, a medication used for the treatment of achondroplasia among children patients, is now available in Georgia, the country’s Ministry of…

Achondroplasia · Ascendis · TransCon CNP

Study Results Promising for Achondroplasia Treatment

Obtained from UBMD Physicians’ Group website. This article was written by Dirk Hoffman and published on November 9, 2023. Teresa Quattrin, MD, UB Distinguished Professor of pediatrics, is a co-author on…

Achondroplasia · BioMarin · Hypochondroplasia · Voxzogo

U.S. Food and Drug Administration Approves BioMarin’s VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia

The following press release was obtained from BioMarin.com. To learn more about BioMarin, VOXZOGO®, vosorotide, and their other pipelines and research for achondroplasia and other skeletal dysplasias, click here. Expanded…