ALLMedicine™ Achondroplasia Center
Research & Reviews 396 results
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10015810
Orphanet Journal of Rare Diseases; Maghnie M, Semler O et. al.
Mar 17th, 2023 - Achondroplasia, caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene, is the most common skeletal dysplasia. The Lifetime Impact of Achondroplasia Study in Europe (LIAISE; NCT03449368) aimed to quantify the burden of acho...
https://clinicaltrials.gov/ct2/show/NCT05145010
Mar 10th, 2023 - This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sp...
https://clinicaltrials.gov/ct2/show/NCT05659719
Mar 3rd, 2023 - The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. The study will include data already c...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9979515
BMC Pediatrics; Xie H, Chen Y et. al.
Mar 2nd, 2023 - Hypochondroplasia (HCH) is a common nonlethal skeletal dysplasia caused by pathogenic variations in the fibroblast growth factor receptor 3 (FGFR3) gene, and HCH has similar clinical manifestations with achondroplasia (ACH), which can be screened ...
https://clinicaltrials.gov/ct2/show/NCT03794609
Mar 2nd, 2023 - This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatment...
Drugs 1 results see all →
Clinicaltrials.gov 32 results
https://clinicaltrials.gov/ct2/show/NCT05145010
Mar 10th, 2023 - This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sp...
https://clinicaltrials.gov/ct2/show/NCT05659719
Mar 3rd, 2023 - The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. The study will include data already c...
https://clinicaltrials.gov/ct2/show/NCT03794609
Mar 2nd, 2023 - This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatment...
https://clinicaltrials.gov/ct2/show/NCT05353192
Feb 28th, 2023 - To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04638153
Feb 21st, 2023 - This is a phase 2 randomized, 3 arm (3 active doses of Recifercept), parallel group dose finding study of safety, tolerability, PK and efficacy The total number of participants is 63 in 2 age straified cohorts of 0-2 years and 6-10 years old. The ...
News 21 results
https://www.medscape.com/viewarticle/972789
Apr 27th, 2022 - In 2021, the Food and Drug Administration approved 50 new drugs, but 24 will not be described here because they would probably not be used in pregnancy. The 24 are Aduhelm (aducanumab) to treat Alzheimer’s disease; Azstarys (serdexmethylphenidate ...
https://www.mdedge.com/obgyn/article/254158/obstetrics/review-new-drugs-may-be-used-during-pregnancy
Gerald G. Briggs, BPharm, FCCP
Apr 26th, 2022 - In 2021, the Food and Drug Administration approved 50 new drugs, but 24 will not be described here because they would probably not be used in pregnancy. The 24 are Aduhelm (aducanumab) to treat Alzheimer’s disease; Azstarys (serdexmethylphenidate.
https://www.medscape.com/viewarticle/963412
Nov 22nd, 2021 - The US Food and Drug Administration (FDA) has approved vosoritide (Voxzogo) daily injection for the treatment of children with achondroplasia, the most common form of human dwarfism. The indication is for children aged 5 years and older who have a...
https://www.medpagetoday.com/endocrinology/growthdisorders/95786
Nov 19th, 2021 - The FDA granted accelerated approval to the first drug to spur growth in kids with the most common form of dwarfism, the agency announced on Friday. Vosoritide (Voxzogo), a once-daily injectable treatment, is indicated for children 5 years and old...
https://www.medscape.com/viewarticle/957381
Aug 30th, 2021 - (Reuters) - U.S.-based Biomarin Pharmaceutical Inc said on Friday the European Commission cleared its treatment for one of the most common forms of dwarfism, the first such approval in the region for a therapy to treat achondroplasia. The approval...