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Novo Nordisk’s Oral Semaglutide Lowers HbA1c in Teens in Pivotal Trial

The positive result sets up pediatric filings in both regions in the second half of 2026.

FDA Expands Tzield Use to Delay Type 1 Diabetes in Children Ages 1 to 7

Injectable Microgel Shows Promise for Reducing Surgical Bleeding in Infants

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

Androgen Signaling Drives Lethal Pediatric Ependymoma, Study Finds

Peer-Reviewed Study Shows Lab-Grown Oesophagus Restores Swallowing in Pigs

Ligand-Based OSM CAR-T Shows Preclinical Efficacy Against Osteosarcoma

Plastic Surgeons Urge Delay of Youth Gender Surgeries as AMA Signals Support

DBV Peanut Allergy Patch Succeeds in Pivotal Phase 3, Setting Up 2026 FDA Filing

FDA Approves One-Time Intrathecal Gene Therapy Itvisma for Older SMA Patients

Exa‑cel Posts Complete Primary-End Point Responses in Early Pediatric Trials, With One Fatal Busulfan Event

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Low-Dose Transplant Drug Preserves Beta Cells in Young People With Type 1 Diabetes, Phase 2 Trial Finds

JAMA Study Finds Donor Heart Valves Grow After Partial Transplant in Children

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

Dual Enzyme Inhibitors Convert Neuroblastoma Tumors into Healthy Neurons in Mice

Psilocybin-Assisted Therapy Shows Early Promise for Treatment-Resistant IBS