<ѻý>Novel Drug Reduced Triglycerides, Risk of Pancreatitis in Persistent Chylomicronemiaѻý> Benefits seen in patients with and without a genetic diagnosis Sep 03, 2024
<ѻý>A Miss for Steroid-Toting Red Blood Cells in Ataxia Telangiectasiaѻý> Phase III trial falls short of efficacy target, but positive signal emerges in younger kids Aug 23, 2024
<ѻý>FDA Advisors Mostly Convinced Arimoclomol Works for Rare Genetic Disorderѻý> If approved, this would be the first drug for Niemann-Pick disease type C Aug 03, 2024
<ѻý>FDA Panel to Consider Arimoclomol for Rare Genetic Disorderѻý> This is the second time the drug has been submitted to the FDA for Niemann-Pick disease type C Aug 01, 2024
<ѻý>Gene-Expression Profiling Can Help Improve Outcomes in Cancers of Unknown Primaryѻý> In randomized study, guided site-specific therapy improved PFS versus empirical chemotherapy Jul 30, 2024
<ѻý>Sotorasib Promising for KRAS–Related Vascular Malformationsѻý> Two patients had rapid symptom improvement and reductions in malformation volume Jul 18, 2024
<ѻý>Gene Therapies for Neovascular AMD Continue to Show Promiseѻý> Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy Jul 18, 2024
<ѻý>Trump's Former Doc Weighs In; 23andMe and Alzheimer's Risk; What's 'Teflon Flu'?ѻý> Health news and commentary gathered by ѻý staff Jul 17, 2024
<ѻý>Psoriasis Joins List of Diseases Tied to Air Pollutionѻý> Poor air quality adds significantly to risk from genetic susceptibility factors Jul 16, 2024
<ѻý>Genes, Air Pollution Combine to Raise Lupus Riskѻý> British data suggest how environmental and genetic factors add together Jul 11, 2024
<ѻý>Can Oligonucleotide Infusions Really Fix Lyme, Cancer, and Other Diseases?ѻý> U.S. clinics market oligonucleotide therapy for a wide range of conditions, without much evidence Jul 05, 2024
<ѻý>Adolescent Obesity Tied to Parents' BMI at the Same Ageѻý> Moderate association between parental and offspring BMI at age 17 Jun 28, 2024
<ѻý>Subcutaneous C5 Inhibitor Approved for PNHѻý> Longer-acting crovalimab designed for more sustained effect and less treatment burden Jun 24, 2024
<ѻý>ALK Inhibitor Shows Promise in Rare, Progressive Tumor Typesѻý> Phase II study found brigatinib exceeded historical control by shrinking 10% of targeted tumors Jun 21, 2024
<ѻý>Gene Therapy May Cure More Rare Diseases, but Drugmakers Have Few Incentivesѻý> Profitability, production cost, and low number of patients are barriers Jun 21, 2024
<ѻý>Alzheimer's Onset Delayed by Rare Mutationѻý> Just one copy of the Christchurch variant conferred protection Jun 19, 2024
<ѻý>Alzheimer's Risk Higher If Mom Had Memory Problemsѻý> Maternal history of memory impairment tied to higher amyloid burden Jun 17, 2024
<ѻý>A Single, Surprising Gene Behind a Disorder That Causes Intellectual Disabilityѻý> Researchers estimate the disorder may affect as many as one in 20,000 young people May 31, 2024
<ѻý>Hereditary Angioedema Attacks Reduced With Donidalorsen Prophylaxisѻý> Investigational treatment passes muster in phase III trial May 31, 2024
<ѻý>Lifestyle-Dementia Links Persist Regardless of Risk Genes, French Study Showsѻý> Dementia prevention efforts may be beneficial despite genetic susceptibility May 23, 2024
<ѻý>Fertility Treatment Safe for Breast Cancer Survivors With BRCA Mutationsѻý> No significant increased risk of recurrence or pregnancy complications May 17, 2024
<ѻý>Gene Editing Improves Vision in Rare and Devastating Retinal Disorderѻý> Therapy improved best corrected visual acuity in four of 14 patients, and others benefited too May 07, 2024
<ѻý>Alzheimer's Variant May Cause Distinct Form of the Diseaseѻý> Almost all APOE4 homozygotes develop Alzheimer's disease May 06, 2024
<ѻý>Impact of Recombinant Replacement Therapy Detailed in Genetic Clotting Disorderѻý> Trial that led to FDA approval shows a big difference in replacement levels versus usual care May 01, 2024
<ѻý>Age-Related Somatic Mutations Implicated in Cardiovascular Deathѻý> Clonal hematopoiesis a red flag for people with asymptomatic carotid atherosclerosis Apr 29, 2024
<ѻý>FDA Approves First Drug for WHIM Syndromeѻý> CXCR4 antagonist mavorixafor effectively boosts neutrophils and lymphocytes to reduce infections Apr 29, 2024
<ѻý>Genetics Studies Have a Diversity Problem That Researchers Struggle to Fixѻý> Most data based on DNA from white people, but small differences can have big health outcomes Apr 28, 2024
<ѻý>Newborn Screening for SMA Appears to Offer Functional Benefitsѻý> German cohort study suggests earlier initiation of treatment benefits sitting, walking Apr 08, 2024
<ѻý>Need 10,000 Daily Steps to Prevent Obesity? It May Depend on Geneticsѻý> Current recommendations may underestimate activity needs for people with high genetic risk Mar 27, 2024
<ѻý>Overdosing on Chemo: A Common Gene Test Could Save Hundreds of Lives Each Yearѻý> Treatment can be deadly for enzyme-deficient patients; NCCN doesn't call for preemptive testing Mar 26, 2024
<ѻý>FDA Approves New Frontline Regimen for Aggressive ALLѻý> Ponatinib lands indication in combination with chemo for Philadelphia chromosome-positive cases Mar 22, 2024
<ѻý>Spinal Delivery of Gene Therapy Promising for Neurodegenerative Diseaseѻý> Early-phase clinical trial was suggestive of slower progression of giant axonal neuropathy Mar 20, 2024
<ѻý>Mutation Linked to Increased Risk of Prostate Cancer Mortalityѻý> Cancer-specific mortality twice as high in patients with specific genotype for HSD3B1 allele Mar 20, 2024
<ѻý>Higher Polygenic Risk Score Tied to Increased Risk for Glaucomaѻý> Risk scores may allow action before irreversible damage occurs in those with ocular hypertension Mar 19, 2024
<ѻý>Cancer Prevention Strategies Linked to Mortality Reduction in BRCA Carriersѻý> Cohort studies indicate oophorectomy, breast MRI surveillance save lives, researchers say Feb 29, 2024
<ѻý>Oral Drug Gains Steam as On-Demand Option for Hereditary Angioedema Attacksѻý> Investigational sebetralstat offered symptom relief in under 2 hours Feb 28, 2024
<ѻý>Faster Cures, More Genetic Treatments: A Q&A With NIH's Joni Rutterѻý> The NCATS Biomedical Data Translator is giving researchers ideas for new disease therapies Feb 23, 2024
<ѻý>Potential Biomarker for Perforated Appendicitis Shows Link to Severe Sepsisѻý> Test that can differentiate between simple and perforated appendicitis is "highly desirable" Feb 20, 2024
<ѻý>Genome Sequencing Identified Variants in Fetal Structural Anomaliesѻý> Researcher touts identification of "high percentage of genetic disorders" Feb 14, 2024
<ѻý>More Patients Rely on Early Prenatal Testing as States Toughen Abortion Lawsѻý> But testing early in pregnancy may be inaccurate Feb 13, 2024