ALLMedicine™ Achondroplasia Center
Research & Reviews 370 results
Bone Stender M, Pimenta JM et. al.
Jun 22nd, 2022 - Achondroplasia (ACH) is a rare, genetic condition and is the most common skeletal dysplasia resulting in disproportionate short stature and numerous multi-systemic comorbidities. As we enter an era of new treatment options which may impact comorbi...
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Orphanet Journal of Rare Diseases; Pesl M, Verescakova H et. al.
Jun 17th, 2022 - Achondroplasia (ACH) is one of the most prevalent genetic forms of short-limbed skeletal dysplasia, caused by gain-of-function mutations in the receptor tyrosine kinase FGFR3. In August 2021, the C-type natriuretic peptide (CNP) analog vosoritide ...
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Orphanet Journal of Rare Diseases; Savarirayan R, Baratela W et. al.
Jun 14th, 2022 - Achondroplasia is associated with disproportionate short stature and significant and potentially severe medical complications. Vosoritide is the first medicine to treat the underlying cause of achondroplasia and data from phase 3 and phase 2 exten...
Journal of Neuroimaging : Official Journal of the America... Calandrelli R, Pilato F et. al.
Jun 13th, 2022 - Whole-spine magnetic resonance imaging (MRI) studies, to identify structural abnormalities associated with the development of symptomatic spinal stenosis in achondroplasia. Forty-two subjects with achondroplasia were grouped into four age-related ...
Advances in Therapy; McGraw SA, Henne JC et. al.
Jun 8th, 2022 - Achondroplasia is characterized by disproportionate short stature accompanied by other changes to the musculoskeletal system. Individuals with this condition typically experience a variety of medical complications. As pharmacologic treatments cont...
Drugs 1 results see all →
Clinicaltrials.gov 30 results
May 23rd, 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 ...
Apr 29th, 2022 - To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
Apr 28th, 2022 - This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.
Apr 18th, 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...
Apr 8th, 2022 - This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be ...
News 19 results
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 ...
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.
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...