ALLMedicine™ Achondroplasia Center
Research & Reviews 392 results
https://doi.org/10.1097/BPO.0000000000002341
Journal of Pediatric Orthopedics; Goldman V, Weiss PL et. al.
Feb 3rd, 2023 - Surgery to achieve long bone lengthening and deformity correction in skeletally immature patients is traditionally performed through external fixators. The incidence of infection during treatment is high. Hydrotherapy is highly beneficial during e...
https://clinicaltrials.gov/ct2/show/NCT04638153
Jan 31st, 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 ...
https://clinicaltrials.gov/ct2/show/NCT04265651
Jan 27th, 2023 - This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in childre...
https://clinicaltrials.gov/ct2/show/NCT05116046
Jan 23rd, 2023 - All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll in this ope...
https://doi.org/10.1111/andr.13385
Andrology Gourinat A, Mazeaud C et. al.
Jan 15th, 2023 - The increase in paternal age and the percentage of births after assisted reproductive technologies (ART) may have consequences on offspring and society's position regarding access to ART must be questioned. Most countries recommend limiting ART to...
Drugs 1 results see all →
Clinicaltrials.gov 32 results
https://clinicaltrials.gov/ct2/show/NCT04638153
Jan 31st, 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 ...
https://clinicaltrials.gov/ct2/show/NCT04265651
Jan 27th, 2023 - This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in childre...
https://clinicaltrials.gov/ct2/show/NCT05116046
Jan 23rd, 2023 - All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll in this ope...
https://clinicaltrials.gov/ct2/show/NCT05598320
Jan 12th, 2023 - The purpose of this clinical trial is to evaluate efficacy and safety of once weekly SC doses of 100 µg CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with geneti...
https://clinicaltrials.gov/ct2/show/NCT04554940
Dec 22nd, 2022 - Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
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...