Genetic Disorders

Two-year data from the pivotal ApproaCH trial showed sustained growth improvements with TransCon CNP in pediatric achondroplasia.

New expert recommendations outline pediatric glucocorticoid dose reduction strategies after starting crinecerfont in classic CAH.

Phase 3 sunRIZE trial of ersodetug in congenital hyperinsulinism missed its primary endpoint but showed CGM-based glycemic gains.

The FDA approved lunsotogene parvec-cwha as the first gene therapy for genetic hearing loss caused by biallelic OTOF variants.

The approval marks the first treatment for this neurodegenerative with a low survival rate beyond 3 years.

The FDA approved fibrinogen, human-chmt to treat acute bleeding episodes in adult and pediatric patients with congenital fibrinogen deficiency.

Genetic testing in at-risk children can improve monitoring, guide activity choices, reduce stress, and prepare families for future therapies.