Achondroplasia, the most common skeletal dysplasia, is characterized by a variety of
medical, functional and psychosocial challenges across the lifespan. The condition is …

Targeting protein-protein interactions (PPIs) has been recently recognized as an emerging
therapeutic approach for several diseases. Up today, more than half a million PPI …

Randomised controlled trials remain the reference standard for healthcare research on
effects of interventions, and the need to report both benefits and harms is essential. The …

The fibroblast growth factor (FGF) family is composed of 18 secreted signaling proteins
consisting of canonical FGFs and endocrine FGFs that activate four receptor tyrosine …

Purpose Achondroplasia is caused by pathogenic variants in the fibroblast growth factor
receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C …

Background Achondroplasia is the most common form of skeletal dysplasia. Recent
advances in therapeutic options have highlighted the need for understanding the burden …

From the medical, pharmaceutical, and social perspectives, 2021 has been a year
dominated by the COVID-19 pandemic. However, despite this global health crisis, the …

Randomised controlled trials often use surrogate endpoints to substitute for a target outcome
(an outcome of direct interest and relevance to trial participants, clinicians, and other …

Randomised controlled trials commonly use surrogate endpoints to substitute for a target
outcome (outcome of direct interest and relevance to trial participants, clinicians, and other …

Achondroplasia is the most common form of human dwarfism. The molecular basis of
achondroplasia was elucidated in 1994 with the identification of the fibroblast growth factor …