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About 1,135 results

ALLMedicine™ Achondroplasia Center

Research & Reviews  392 results

Hydrotherapy for Patients With External Fixation: Effect on Infectious Events.
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...

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
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 ...

Study of Infigratinib in Children With Achondroplasia
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...

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
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...

Impact of paternal age on assisted reproductive technology outcomes and offspring healt...
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...

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Drugs  1 results see all →

Clinicaltrials.gov  32 results

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
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 ...

Study of Infigratinib in Children With Achondroplasia
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...

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
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...

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
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...

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
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

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News  21 results

Review of New Drugs That May Be Used During Pregnancy
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 ...

Review of new drugs that may be used during pregnancy
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.

FDA Approves Vosoritide for Children With Achondroplasia
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...

FDA OKs First Drug for Most Common Form of Dwarfism
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...

Europe Approves Biomarin Drug for Type of Dwarfism in Region's First
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...

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Patient Education  8 results see all →