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- Dr. Rui Yang of Texas Children's Hospital and Baylor College of Medicine received the Burroughs Wellcome Fund Career Award for Medical Scientists, providing $700,000 over five years. - The award supports research into molecular and cellular mechanisms underlying rare genetic immune disorders, aiming to identify novel disease pathways and therapeutic targets. - Yang's work seeks to uncover new genetic diseases and leverage ultra-rare disorders to illuminate fundamental human immune biology for both rare and common conditions. - The CAMS program supports physician-scientists transitioning to independent academic careers, with Yang continuing as a tenure-track assistant professor in allergy and immunology.
- A multicenter randomized controlled trial across 13 pediatric transplant centers evaluated the Medication Level Variability Index (MLVI) to flag adolescents and young adults at elevated risk for liver transplant rejection due to medication nonadherence. - The study screened over 3,000 health records and enrolled 148 high-risk participants, finding that routine MLVI use substantially reduced overall rejection rates to levels typically seen in low-risk patients. - Patients receiving a two-year remote behavioral intervention experienced approximately half as many rejection-related events and re-transplants compared to those receiving standard care. - Researchers strongly support incorporating MLVI into routine clinical practice, enabling a shift from reacting to rejection to preventing it using data already available in electronic health records.
- Researchers at Baylor College of Medicine identified a core 12-gene presymptomatic signature disrupted exclusively in MECP2-mutant brain cells in mouse models of Rett syndrome. - The study revealed that trilaminar interneurons, a previously unrecognized cell type in Rett syndrome, exhibit the most severe transcriptional dysregulation when MeCP2 function is lost. - By physically separating healthy and mutant cells from mosaic female brains, scientists discovered that genetically normal neurons are also affected by neighboring defective cells through non-cell-autonomous effects. - The findings, published in Science Advances, provide objective biomarkers for monitoring therapeutic efficacy and suggest early intervention targets to slow or prevent disease progression.
- Dr. Jimmy Holder and his team at Texas Children's Hospital and Baylor College of Medicine have received up to $17.25 million from ARIA to join the IMPACT Network. - The IMPACT Network comprises 12 collaborative sites worldwide aimed at accelerating clinical trial readiness for autism therapies, including rare genetic and non-genetic forms. - The team will contribute to a short-term natural history and clinical endpoint study focused on children with profound autism. - Holder's lab focuses on SHANK3 and SYNGAP1 genes, which are linked to severe neurodevelopmental disorders including autism spectrum disorders and intellectual disabilities.
- Researchers identified a novel genetic disorder caused by biallelic loss-of-function variants in the TMEM63B gene, presenting as severe childhood interstitial lung disease in five individuals from four unrelated families. - Unlike heterozygous gain-of-function TMEM63B variants linked to epilepsy and developmental delays, biallelic loss-of-function variants result in early-onset respiratory distress and lung abnormalities without epilepsy. - Functional studies confirmed a loss-of-function mechanism, with patient phenotypes mirroring Tmem63b-knockout mice that exhibit neonatal respiratory failure. - The discovery, facilitated by the NIH-funded Undiagnosed Diseases Network and international collaboration, offers crucial diagnostic answers for affected families and may significantly impact management of this rare surfactant-related disorder.
- Neurogene reported positive interim data from its Phase 1/2 trial of NGN-401, showing all eight pediatric participants gained developmental milestones across core domains of Rett syndrome with durable improvements lasting up to 24 months. - The company has dosed multiple participants in its Embolden registrational trial in Q4 2025 and expects to complete dosing in Q2 2026, with interim data from both pediatric and adolescent/adult cohorts planned for mid-2026. - NGN-401 at the 1E15 vg dose continues to demonstrate a favorable safety profile with no evidence of hemophagocytic lymphohistiocytosis (HLH), positioning the therapy as a potential best-in-class treatment for Rett syndrome.
- The National Institutes of Health has awarded Baylor College of Medicine $15.6 million to fund two rare disease research consortia over five years. - The Brittle Bone Disorders Consortium receives $7.4 million to continue studying osteogenesis imperfecta, having already recruited the world's largest longitudinal cohort of over 1,000 patients. - The newly launched Rare Organic Acidemias Research Consortium receives $8.2 million to conduct clinical trials and observational studies on genetic metabolic disorders affecting protein and energy metabolism. - Both consortia will focus on developing new therapies, training investigators, and collaborating with patient advocacy organizations to improve treatment outcomes.
- AstraZeneca's FluMist Home is now available for home delivery in 34 US states, marking the first time Americans can self-administer a flu vaccine at home. - The nasal spray vaccine can be used by adults under 50 and children as young as 2 with parental assistance, following FDA approval for self-administration in September 2024. - The home delivery option aims to address low vaccination rates, as less than half of Americans received flu vaccines during the severe 2024-2025 season that caused up to 82 million illnesses. - AstraZeneca's usability study demonstrated 100% of intended users successfully administered a full dose without healthcare provider supervision.
- The first commercial gene therapy treatments ever delivered directly to the brain in the United States have been successfully performed using ClearPoint Neuro's SmartFlow Neuro Cannula to administer KEBILIDI for AADC deficiency. - KEBILIDI (eladocagene exuparvovec-tneq) is delivered directly to the putamen region of the brain through stereotactic neurosurgery, with the SmartFlow cannula being the only FDA-authorized device for this administration. - ClearPoint Neuro reported record clinical activity in July with 17 global patients treated across 11 different drug platforms, while nine of the company's partners have now been accepted into FDA expedited review programs. - New ICD-10 codes specific to neuro infusions will become effective October 1, 2025, to assist in tracking commercial use of brain-delivered therapies.
- Two innovative clinical trials are advancing treatment options for acute myeloid leukemia (AML), targeting both newly diagnosed pediatric patients and those with relapsed or refractory disease. - The UPDATE AML study is testing new chemotherapy combinations including venetoclax-based regimens in children and young adults, aiming to maintain survival while reducing long-term side effects. - A phase 1/2 trial is evaluating CD33 CAR-NK cell therapy from healthy donors for patients with relapsed or refractory AML, representing a novel immunotherapy approach. - Both studies incorporate advanced diagnostic techniques including personalized genetic profiling and residual disease detection to improve treatment monitoring and outcomes.
