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About 813 results

ALLMedicine™ Achondroplasia Center

Research & Reviews  295 results

Study of Infigratinib in Children With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04265651

Jan 20th, 2022 - 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...

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

Jan 19th, 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...

Disease-specific complications and multidisciplinary interventions in achondroplasia.
https://doi.org/10.1007/s00774-021-01298-z 10.1038/s41436-021-01287-7
Journal of Bone and Mineral Metabolism; Kitoh H, Matsushita M et. al.

Jan 15th, 2022 - Achondroplasia (ACH) is the most common skeletal dysplasia and characterized by a disproportionate short stature, macrocephaly with frontal bossing, exaggerated lumbar lordosis, and trident hands. It is induced by activated mutations in the fibrob...

Genetics of Achondroplasia
https://emedicine.medscape.com/article/941280-overview

Jan 10th, 2022 - Background Achondroplasia, a nonlethal form of chondrodysplasia, is the most common type of short-limb dwarfism. This skeletal dysplasia is inherited as a Mendelian autosomal dominant trait with complete penetrance. Approximately 80% of cases are ...

Impact of achondroplasia on Latin American patients: a systematic review and meta-analy...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8728937
Orphanet Journal of Rare Diseases; Fano V, Kim CA et. al.

Jan 6th, 2022 - Achondroplasia (ACH), the most common form of disproportionate short stature, is caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene. Recent advances in drug therapy for ACH have highlighted the importance of elucidating...

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Drugs  1 results see all →

Clinicaltrials.gov  8 results

Study of Infigratinib in Children With Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT04265651

Jan 20th, 2022 - 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...

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

Jan 19th, 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...

The Norwegian Adult Achondroplasia Study
https://clinicaltrials.gov/ct2/show/NCT03780153

Dec 15th, 2021 - The present study aims to investigate the degree and extent of spinal stenosis, chronic pain, sleep apnoea, impaired hearing, cardiovascular risk factors and body composition in Norwegian adults with achondroplasia. The study will also explore the...

Lifetime Impact of Achondroplasia Study in Europe-LIAISE
https://clinicaltrials.gov/ct2/show/NCT03449368

Apr 5th, 2021 - This is a multinational, epidemiological, observational, retrospective, cross-sectional study of individuals with achondroplasia (subjects). This study will be conducted at up to approximately 20 sites in European countries. Subjects will be invit...

Lifetime Impact Study for Achondroplasia
https://clinicaltrials.gov/ct2/show/NCT03872531

Apr 5th, 2021 - This is a multinational, epidemiological, observational, retrospective, cross-sectional study of individuals with achondroplasia (subjects). This study will be conducted at up to approximately 4 sites in Latin American Countries - Brazil, Argentin...

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News  17 results

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...

FDA Approves Infigratinib for FGFR2+ Cholangiocarcinoma
https://www.onclive.com/view/fda-approves-infigratinib-for-cholangiocarcinoma

May 28th, 2021 - The FDA has approved infigratinib (Truseltiq) for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement.1 The application for infigratinib was evaluated unde...

Success for Vosoritide Spurs Testing in More Growth Disorders
https://www.medscape.com/viewarticle/947834

Mar 22nd, 2021 - On the basis of the quality of sustained bone growth achieved with vosoritide in dwarfism, studies are underway or being considered for more diseases that impair bone growth, according to discussion that followed the presentation of a phase 3 tria...

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Patient Education  4 results see all →