NATIONWIDE CHILDREN'S HOSPITAL

MAIA Biotechnology, Inc. announced FDA's Rare Pediatric Disease designation for THIO in treating pediatric-type diffuse high-grade gliomas (PDHGG). THIO, an anti-cancer agent, activates the immune system and evades tumor immunosuppression. The designation offers potential for a priority review voucher, valued at $100 million, upon FDA approval.

MAIA Biotechnology announced FDA's Rare Pediatric Disease designation for THIO, its anti-cancer agent for pediatric-type diffuse high-grade gliomas. THIO activates the immune system and evades tumor immunosuppression, with potential for priority review vouchers upon FDA approval.

MAIA Biotechnology announced FDA's Rare Pediatric Disease Designation for THIO, its investigational drug for pediatric-type diffuse high-grade gliomas. THIO, with orphan drug designations for hepatocellular carcinoma, small-cell lung cancer, and glioblastoma, is in phase II trials for various cancers. MAIA's collaboration with Nationwide Children's Hospital demonstrated THIO's effectiveness with ionizing radiation in treating diffuse intrinsic pontine glioma, presented at AACR 2024. THIO activates innate and adaptive immune responses, offering a new approach to pediatric cancer treatment.

Fortress Biotech announces FDA approval for Emrosi to treat rosacea and potential approval for cosibelimab for cutaneous squamous cell carcinoma, with financial results and recent corporate highlights for Q3 2024.

A study finds a 50% increase in psychology referrals for children with food allergies from 2018 to 2023, driven by anxiety related to potential accidental ingestion and anaphylaxis.

Delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy missed primary endpoint in EMBARK trial but showed differences in secondary outcomes, including clinically relevant improvements in time to rise and 10-meter walk/run. FDA granted full approval based on totality of evidence, despite primary endpoint failure.

A global study identified molecular predictors of survival among Black AML patients, highlighting the need for ancestry-specific genetic factors in AML risk layers and clinical trials. The study found that 73% of recurrent gene mutations in Black patients were unique or rare in white patients, affecting disease-free survival and overall survival. Ancestry-specific mutations altered risk group assignment for one-third of Black patients, improving outcome predictions.