ALLMedicine™ Achondroplasia Center

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...

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...

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,

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...

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...

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 ...

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...

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...

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...

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.

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 ...

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.

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...

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...

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...