ALLMedicine™ Dwarfism Center

May 14th, 2022 - To explore the experiences of women of short stature, including women with dwarfism and osteogenesis imperfecta (OI), during the perinatal period. Qualitative descriptive. Telephone interviews with women of short stature from across the United Sta...

May 3rd, 2022 - A wide variety of medications have been associated with lichenoid drug eruption. They present similarly or even identically to idiopathic lichen planus, both clinically and histologically. Lichenoid eruption has been associated with recombinant hu...

Apr 16th, 2022 - Microcephalic Osteodysplastic Primordial Dwarfism (MOPD) Type II is an autosomal recessive condition encompassing a heterogeneous group of disorders characterized by symmetrical growth retardation leading to dwarfism, microcephaly, and a range of ...

Mar 26th, 2022 - The chondrocyte-specific miR-140 miRNAs are necessary for normal endochondral bone growth in mice. miR-140 deficiency causes dwarfism and craniofacial deformity. However, the physiologically important targets of miR-140 miRNAs are still unclear. T...

Mar 18th, 2022 - We report a patient with microcephalic primordial dwarfism with predominant Meier-Gorlin syndrome phenotype with ichthyosis and disabling multiple joint deformities in addition to classic features of the syndrome. The patient was a 10.5-year-old g...

Mar 17th, 2022 - Mucopolysaccharidosis IVA (MPS IVA, Morquio A Disease) is a rare autosomal recessive disorder caused by a deficiency of the lysosomal enzyme, N-acetylgalactosamine 6-sulfate sulfatase (GALNS). GALNS catalyzes the degradation of the glycosaminoglyc...

Apr 9th, 2021 - The mucopolysaccharidoses are a group of inherited lysosomal storage disorders. Lysosomes function as the primary digestive units within cells. Enzymes within lysosomes break down or digest particular nutrients, such as certain carbohydrates and f...

Sep 29th, 2020 - The goal of this registry is to collect information on individuals with Microcephalic Osteodysplastic Primordial Dwarfism Type II (also called MOPDII) and other forms of microcephalic primordial dwarfism. The registry will enable detailed natural ...

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...

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...

Oct 26th, 2020 - A year of daily subcutaneous vosoritide was associated with a 0.6-inch (1.5-cm) greater increase in height than placebo in children with achondroplasia, the most common form of human dwarfism. There was no difference in the incidence of side effec...