Gene-Editing Therapy Reduced Attacks in Hereditary Angioedema Single infusion also reduced total plasma kallikrein protein levels versus placebo Oct 24, 2024
First Cases of New Neurology Syndrome Identified Long non-coding RNA implicated in human disease Oct 23, 2024
Evidence of Durable Vision Improvement With Gene Therapy for Retinitis Pigmentosa Significant improvement beyond 100 weeks after single injection of mutation-agnostic MCO-010 Oct 22, 2024
Gene Therapy Details Revealed in Duchenne Trial Differences between delandistrogene moxeparvovec and placebo emerge in secondary outcomes Oct 18, 2024
Allele May Heighten Risk of Severe Ulcerative Colitis Finding on HLA-DRB1*01:03 supports need for more and earlier genetic studies Oct 15, 2024
Elamipretide for Barth Syndrome Survives FDA Advisory Vote Panelists acknowledge difficulty generating stronger data in ultra-rare disease Oct 11, 2024
Gene Therapy for Rare Brain Disease Effective, but Comes With Blood Cancer Risk 10% of patients in trials leading to eli-cel's approval developed MDS or AML Oct 09, 2024
FDA Reviewers Cast Doubt on Drug Candidate for Barth Syndrome Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease Oct 09, 2024
Hormonal Contraception Linked to Increased Breast Cancer Risk in BRCA1 Carriers Higher risk for ever use, increasing duration of use but no increase in BRCA2 carriers Oct 07, 2024
Second Drug OK'd for Ultra-Rare Neurodegenerative Disease FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C Sep 25, 2024
First Drug Approved for Niemann-Pick Disease Type C Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder Sep 20, 2024
New Genetic Analysis of Animals in Wuhan Market in 2019 May Help Find COVID's Origin Findings "shift the dial more in favor of an animal origin," said one expert Sep 19, 2024
RFK Jr. Helping Trump Pick CDC Chief; Excess COVID Death Lingers; Turmoil at 23andMe Health news and commentary gathered by ľֱ staff Sep 19, 2024
Novel Drug Reduced Triglycerides, Risk of Pancreatitis in Persistent Chylomicronemia Benefits seen in patients with and without a genetic diagnosis Sep 03, 2024
A Miss for Steroid-Toting Red Blood Cells in Ataxia Telangiectasia Phase III trial falls short of efficacy target, but positive signal emerges in younger kids Aug 23, 2024
FDA Advisors Mostly Convinced Arimoclomol Works for Rare Genetic Disorder If approved, this would be the first drug for Niemann-Pick disease type C Aug 03, 2024
FDA Panel to Consider Arimoclomol for Rare Genetic Disorder This is the second time the drug has been submitted to the FDA for Niemann-Pick disease type C Aug 01, 2024
Gene-Expression Profiling Can Help Improve Outcomes in Cancers of Unknown Primary In randomized study, guided site-specific therapy improved PFS versus empirical chemotherapy Jul 30, 2024
Sotorasib Promising for KRAS–Related Vascular Malformations Two patients had rapid symptom improvement and reductions in malformation volume Jul 18, 2024
Gene Therapies for Neovascular AMD Continue to Show Promise Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy Jul 18, 2024
Trump's Former Doc Weighs In; 23andMe and Alzheimer's Risk; What's 'Teflon Flu'? Health news and commentary gathered by ľֱ staff Jul 17, 2024
Psoriasis Joins List of Diseases Tied to Air Pollution Poor air quality adds significantly to risk from genetic susceptibility factors Jul 16, 2024
Genes, Air Pollution Combine to Raise Lupus Risk British data suggest how environmental and genetic factors add together Jul 11, 2024
Can Oligonucleotide Infusions Really Fix Lyme, Cancer, and Other Diseases? U.S. clinics market oligonucleotide therapy for a wide range of conditions, without much evidence Jul 05, 2024
Adolescent Obesity Tied to Parents' BMI at the Same Age Moderate association between parental and offspring BMI at age 17 Jun 28, 2024
Subcutaneous C5 Inhibitor Approved for PNH Longer-acting crovalimab designed for more sustained effect and less treatment burden Jun 24, 2024
ALK Inhibitor Shows Promise in Rare, Progressive Tumor Types Phase II study found brigatinib exceeded historical control by shrinking 10% of targeted tumors Jun 21, 2024
Gene Therapy May Cure More Rare Diseases, but Drugmakers Have Few Incentives Profitability, production cost, and low number of patients are barriers Jun 21, 2024
Alzheimer's Onset Delayed by Rare Mutation Just one copy of the Christchurch variant conferred protection Jun 19, 2024
Alzheimer's Risk Higher If Mom Had Memory Problems Maternal history of memory impairment tied to higher amyloid burden Jun 17, 2024
A Single, Surprising Gene Behind a Disorder That Causes Intellectual Disability Researchers estimate the disorder may affect as many as one in 20,000 young people May 31, 2024
Hereditary Angioedema Attacks Reduced With Donidalorsen Prophylaxis Investigational treatment passes muster in phase III trial May 31, 2024
Lifestyle-Dementia Links Persist Regardless of Risk Genes, French Study Shows Dementia prevention efforts may be beneficial despite genetic susceptibility May 23, 2024
Fertility Treatment Safe for Breast Cancer Survivors With BRCA Mutations No significant increased risk of recurrence or pregnancy complications May 17, 2024
Gene Editing Improves Vision in Rare and Devastating Retinal Disorder Therapy improved best corrected visual acuity in four of 14 patients, and others benefited too May 07, 2024
Alzheimer's Variant May Cause Distinct Form of the Disease Almost all APOE4 homozygotes develop Alzheimer's disease May 06, 2024
Impact of Recombinant Replacement Therapy Detailed in Genetic Clotting Disorder Trial that led to FDA approval shows a big difference in replacement levels versus usual care May 01, 2024
Age-Related Somatic Mutations Implicated in Cardiovascular Death Clonal hematopoiesis a red flag for people with asymptomatic carotid atherosclerosis Apr 29, 2024
FDA Approves First Drug for WHIM Syndrome CXCR4 antagonist mavorixafor effectively boosts neutrophils and lymphocytes to reduce infections Apr 29, 2024
Genetics Studies Have a Diversity Problem That Researchers Struggle to Fix Most data based on DNA from white people, but small differences can have big health outcomes Apr 28, 2024