<ѻý>A Clue on Why Lupus, Other Autoimmune Diseases Strike Far More Women Than Menѻý> New research shows that the extra X chromosome is involved, but in an unexpected way Feb 01, 2024
<ѻý>Novel Gene Therapy Allows Kids With Inherited Deafness to Hearѻý> "This is real proof showing gene therapy is working," says researcher Jan 25, 2024
<ѻý>Cost of Genomic Testing on the Minds of Many Oncologistsѻý> Insurance coverage, patient out-of-pocket costs key considerations for 80% of survey respondents Jan 16, 2024
<ѻý>MS Risk Linked to Ancient Migrationsѻý> Findings may help demystify multiple sclerosis Jan 10, 2024
<ѻý>Retinal Tumor Risk Incidentally Detected at 29 Weeks' Gestationѻý> Finding poses clinical and ethical dilemma for parents and clinicians Jan 08, 2024
<ѻý>FDA Approves New Agent for Nerve Pain From Rare Inherited Diseaseѻý> Eplontersen gets a nod for polyneuropathy from hereditary transthyretin-mediated amyloidosis Dec 22, 2023
<ѻý>Pomalidomide Cuts Severe Epistaxis in Genetic Bleeding Disorderѻý> 24 weeks of daily treatment significantly reduced recurrent nosebleeds in HHT trial Dec 13, 2023
<ѻý>Why Is VEXAS Syndrome Often Fatal? Here's an Answerѻý> New study suggests immunodeficiency is a previously unrecognized feature Dec 12, 2023
<ѻý>A Closer Look at the Approval of CRISPR/Cas9 Gene Therapy for Sickle Cell Diseaseѻý> This landmark event brings up broader considerations and implications for other diseases Dec 11, 2023
<ѻý>Antisense Drug Trial Provides Key Insights About Huntington's Diseaseѻý> Lessons learned from investigational tominersen Dec 06, 2023
<ѻý>Jamie Foxx's Health Scare; 23andMe Hacked; Decongestant Probe Announcedѻý> Health news and commentary from around the Web gathered by ѻý staff Dec 05, 2023
<ѻý>Here's Why People Think They Developed Food Allergiesѻý> Survey finds that 13% thought genetics were responsible, 27% blamed eating behaviors Nov 16, 2023
<ѻý>Promising Debut for CRISPR Gene Editing Therapy for LDL Loweringѻý> Preliminary data support concept of "molecular surgery" on PCSK9 gene Nov 12, 2023
<ѻý>Zoonotic Spillovers; DNA and RNA Testing for Cancer Riskѻý> Also in TTHealthWatch: genes and health conditions among Icelanders Nov 11, 2023 podcast
<ѻý>First Treatment Approved for Genetic Clotting Disorderѻý> Adzynma indicated as prophylactic or on-demand enzyme replacement therapy in cTTP Nov 10, 2023
<ѻý>Moving Beyond the 'Angelina Jolie Effect' to Address Cancer Disparitiesѻý> A "local heroes effect" can better reach communities of color Nov 09, 2023
<ѻý>Potential Cure for Sickle Cell Clears Hurdle for Approvalѻý> FDA advisors suggest the benefits of gene therapy outweigh risks of off-target editing Oct 31, 2023
<ѻý>Quick Genetic Test Offers Hope for Sick, Undiagnosed Kids, but Few Insurers Cover Itѻý> Test can "change the trajectory of these children's diseases" Oct 29, 2023
<ѻý>FDA Turns Down ATTR Cardiomyopathy Drugѻý> Decision cites insufficient evidence of clinical meaningfulness Oct 09, 2023
<ѻý>In BRCA Breast Cancer, Prophylactic Salpingo-Oophorectomy Tied to Better Survivalѻý> PSO should be performed early, particularly for those with the BRCA1 variant, researchers say Oct 04, 2023
<ѻý>FDA Greenlights Test for Cancer-Predisposing Genetic Mutationsѻý> First-of-its-kind DNA test achieved 99% accuracy for variants in dozens of genes Oct 03, 2023
<ѻý>FDA OKs Another Injectable for Rare Kidney Disorderѻý> Nedosiran approved for patients ages 9 years and older with primary hyperoxaluria type 1 Oct 02, 2023
<ѻý>New Option OK'd for Pompe Diseaseѻý> FDA approves two-component therapy for patients not improving on enzyme replacement therapy Sep 29, 2023
<ѻý>Huntington's Disease More Severe in Black Patients at Baselineѻý> Outreach to families of minority patients may help identify genetic disorder Sep 14, 2023
<ѻý>FDA Panel Reluctantly Backs Patisiran for ATTR Cardiomyopathyѻý> Clinical meaningfulness is a different question, however Sep 13, 2023
<ѻý>Some Sinus Relief for Cystic Fibrosis Patients on Trikaftaѻý> Lung function, BMI, bacterial swabs, sweat chloride levels also improved Sep 11, 2023
<ѻý>Rapid Genome Sequencing in Infants With Epilepsy Shows Immediate Benefitѻý> Pilot study suggests early testing for seizures, regardless of treatment setting Sep 07, 2023
<ѻý>Ultra-Rare Immune System Disease Gets First FDA-Approved Treatmentѻý> Pozelimab reduced hospitalizations, albumin transfusions in CHAPLE disease patients Aug 18, 2023
<ѻý>Gene Therapy Promising for Rare Jaundice Syndromeѻý> Early-phase data suggest a dose that can keep bilirubin in check without hours of phototherapy Aug 16, 2023
<ѻý>Cautious Return-to-Play Appears Safe for Athletes With Heart Conditionsѻý> Shift away from blanket restrictions is not placing top-tier athletes at undue risk, data suggest Aug 14, 2023
<ѻý>New Recommendations Address Global Inequality in Sickle Cell Disease Careѻý> Commission lays out strategies for progress by 2025 and beyond Jul 13, 2023
<ѻý>Whole Genome Sequencing Boosts Diagnosis of Rare Disease in Infantsѻý> Whole genome scans took longer than a commercially available test, but not for most urgent cases Jul 11, 2023
<ѻý>MS Disease Severity Tied to Genetic Variantѻý> Findings mark a significant breakthrough in understanding how MS progresses Jun 28, 2023
<ѻý>FDA Staff Weighs 'Alternative' Data on Retinoid for Rare Bone Disorderѻý> Challenges of drug development in fibrodysplasia ossificans progressiva on full display Jun 27, 2023
<ѻý>Bicuspid Aortic Valves and Aortopathy Cluster Among Close Relativesѻý> Large study supports wider screening of family members of probands Jun 15, 2023
<ѻý>High-Dose Leptin Reversed Severe Genetic Obesity in Two Patientsѻý> Metreleptin doses several times higher than recommended brought weight down to near normal Jun 14, 2023
<ѻý>Cautious Hope for Endovascular Aortic Tx in Connective Tissue Diseaseѻý> Success observed in usually contraindicated Marfan, Loeys-Dietz syndromes Jun 14, 2023
<ѻý>Full-On Reversal of Cardiac Amyloidosis Possible With Antibodiesѻý> Future treatments don't have to be limited to slowing disease, case series suggests Jun 07, 2023
<ѻý>How to Overcome the Catch-22 in Precision Medicine Researchѻý> Improving the patient experience will go a long way May 31, 2023
<ѻý>Rare Skin Blistering Condition Gets First Drug Approvedѻý> FDA's decision marks first approval for a redosable gene therapy May 19, 2023