Publications by authors named "R Savarirayan"
Achondroplasia is the most common genetic form of short-limbed skeletal dysplasia (dwarfism). Clinical manifestations and complications can affect individuals across the lifespan, including the need for adaptations for activities of daily living, which can affect quality of life. Current international guidelines focus on symptomatic management, with little discussion regarding potential medication, as therapeutic options were limited at the time of their publication.
View Article and Find Full Text PDFBackground: Vosoritide is a C-type natriuretic peptide analog that addresses an underlying pathway causing reduced bone growth in achondroplasia. Understanding the vosoritide treatment effect requires evaluation over an extended duration and comparison with outcomes in untreated children.
Methods: After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.
Article Synopsis
- Achondroplasia is a genetic condition that leads to significant short stature and various medical challenges, and infigratinib is a new treatment being tested for children affected by this disorder.
- A phase 2 study involved 72 children aged 3 to 11, testing varying doses of infigratinib over 6 months, with adjustments possible during a further 12-month period, focusing on the safety and height growth of the participants.
- Results indicated that all participants experienced mild to moderate side effects, but none stopped treatment; most significantly, children in the highest dose group showed a notable increase in height growth compared to baseline, suggesting the treatment could be effective.
Article Synopsis
- * The workshop aimed to address the lack of understanding among physicians about the pathophysiology, radiology, and orthopedic options for managing long bone deformities in children with achondroplasia.
- * The event featured presentations from a multinational survey, lectures, a debate, and interactive discussions, attracting 150 participants from 71 cities across 31 countries.