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About 1,068 results

ALLMedicine™ Achondroplasia Center

Research & Reviews  375 results

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04638153

Aug 5th, 2022 - 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 ...

Achondroplasia Natural History Multicenter Clinical Study
https://clinicaltrials.gov/ct2/show/NCT02597881

Aug 1st, 2022 - The purpose of this protocol is to create an electronic registry to house phenotypic information from patients with all types of bone conditions. The initial focus of this registry will be to include U.S. patients with achondroplasia. Once populat...

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04085523

Aug 1st, 2022 - The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.

Myasthenia gravis complicating the surgical management of achondroplasia: a case-based ...
https://doi.org/10.1007/s00381-022-05617-1
Child's Nervous System : ChNS : Official Journal of the I... Afshari FT, Parida A et. al.

Jul 31st, 2022 - Achondroplasia is the commonest skeletal dysplasia of autosomal dominant inheritance caused by "gain of function" mutations in the fibroblast growth factor receptor 3 (FGFR3) gene. Foramen magnum compression due to accelerated ossification and spi...

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT05116046

Jul 29th, 2022 - 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...

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Drugs  1 results see all →

Clinicaltrials.gov  30 results

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04638153

Aug 5th, 2022 - 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 ...

Achondroplasia Natural History Multicenter Clinical Study
https://clinicaltrials.gov/ct2/show/NCT02597881

Aug 1st, 2022 - The purpose of this protocol is to create an electronic registry to house phenotypic information from patients with all types of bone conditions. The initial focus of this registry will be to include U.S. patients with achondroplasia. Once populat...

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04085523

Aug 1st, 2022 - The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT05116046

Jul 29th, 2022 - 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 Safety of Vosoritide in At-risk Infants With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04554940

Jul 28th, 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  19 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  2 results see all →