Rezultate promițătoare pentru un tratament destinat unei boli rare de creștere la copii • Newsweek România

A new advanced clinical trial treatment shows promise for children with a rare genetic growth disorder, significantly improving bone development. In a phase III study, children receiving the treatment grew 2.33 centimeters more annually than those on placebo. The results were published in NEJM Evidence and presented at the European Society of Pediatric Endocrinology Congress. Hypochondroplasia, a rare skeletal dysplasia, affects bone growth and can lead to various complications. Currently, no medications are approved by the EMA or FDA for this condition.