About 1,115 results

ALLMedicine™ Achondroplasia Center

Research & Reviews  386 results

Clinical manifestations and outcomes of 20 Korean hypochondroplasia patients with the F...
Experimental and Clinical Endocrinology & Diabetes : Offi... Kim HY, Lee YA et. al.

Nov 29th, 2022 - Hypochondroplasia is a skeletal dysplasia caused by activating pathologic variants of FGFR3. The N540K variant accounts for 60%-70% of reported cases and is associated with severe manifestations. Here, we analyzed the clinical manifestations and o...

A 66-Year-Old Woman with Achondroplasia and Quadriparesis with an Ovarian Cyst Erroneou...
The American Journal of Case Reports; Chigrinskiy P, Heyman SN et. al.

Oct 28th, 2022 - BACKGROUND Automated bladder scanning has become a principal tool in the assessment and management of chronically debilitated patients residing in skilled nursing facilities, hospices, and acute inpatient settings. To a large extent, the bladder s...

Foramen magnum stenosis in a 5-month-old boy with achondroplasia.
CMAJ : Canadian Medical Association Journal = Journal De ... Malhotra AK, Parker W et. al.

Oct 21st, 2022 - Foramen magnum stenosis in a 5-month-old boy with achondroplasia.|2022|Malhotra AK,Parker W,Dirks PB,|diagnostic imaging,diagnostic imaging,complications,diagnostic imaging,

Evaluation of Volumetric Bone Mineral Density, Bone Microarchitecture, and Bone Strengt...
Calcified Tissue International; Liang H, Qi W et. al.

Oct 20th, 2022 - Achondroplasia (ACH) is a skeletal disorder caused by fibroblast growth factor receptor 3 (FGFR3) variants. Volumetric bone mineral density (vBMD), bone microarchitecture, and strength have not been evaluated in these patients previously. This stu...

Epidemiology of Eustachian tube dysfunction and related otologic diagnoses among childr...
International Journal of Pediatric Otorhinolaryngology; Dorney I, Otteson T et. al.

Oct 19th, 2022 - To investigate the prevalence of diagnosed Eustachian tube dysfunction and related otologic diagnoses among children with achondroplasia as compared to a control population. The TriNetX Analytics Network, a federated health research network that a...

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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

Oct 12th, 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 ...

Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Oct 10th, 2022 - 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...

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

Sep 14th, 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...

Achondroplasia Natural History Multicenter Clinical Study

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

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.

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News  21 results

Review of New Drugs That May Be Used During Pregnancy

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
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

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

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

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 →