<ѻý>Topical Gene Therapy Heals Blistering Skin Disease Lesionsѻý> Randomized trial shows "dramatic" benefits in rare genetic disease Dec 14, 2022
<ѻý>Assay Predicts Breast Cancer Patients Who Will Benefit From Ovarian Suppressionѻý> Low score suggests improved outcomes with ovarian suppression plus adjuvant endocrine therapy Dec 07, 2022
<ѻý>Decoding Cancerѻý> A conversation with Harold Varmus, MD, who received the Nobel Prize in Physiology or Medicine in 1989 Dec 06, 2022 podcast
<ѻý>Chris Hemsworth's APOE4 Alzheimer's Geneѻý> The Thor actor recently learned about his genetic predisposition Dec 01, 2022
<ѻý>FDA OKs First Gene Therapy for Hemophilia Bѻý> Hemgenix reduced rate of annual bleeds, eliminated need for factor IX prophylaxis therapy Nov 22, 2022
<ѻý>Early Cardiac Amyloidosis Still Bad News Without Treatmentѻý> Observational study supports treating even asymptomatic patients Nov 16, 2022
<ѻý>CRISPR Shows Promise for Hereditary Angioedema in First-in-Human Trialѻý> No safety concerns reported and swelling attacks declined Nov 15, 2022
<ѻý>Promising Results in First Test of In Utero Enzyme-Replacement Therapyѻý> Treatment was safe, effective in one baby with infantile-onset Pompe disease Nov 09, 2022
<ѻý>My Risk Revealed: A Confirmed BRCA2 Mutationѻý> My dad's cancer diagnosis offered me invaluable information Oct 30, 2022
<ѻý>Texas Says DNA Kits for Schoolchildren Not Tied to Uvalde Shootingѻý> State education agency says effort was designed to help law enforcement find missing kids Oct 27, 2022
<ѻý>Gene Therapy for Degenerative Brain Disorder Wins FDA Nodѻý> $3 million price tag for one-time treatment Sep 19, 2022
<ѻý>Blood Type Tied to Early-Onset Stroke Riskѻý> Findings point to prothrombotic mechanisms in younger stroke patients Sep 01, 2022
<ѻý>Pralsetinib Shows Promise in Diverse RET Fusion-Positive Cancersѻý> An ORR of 57% supports results previously observed in NSCLC and thyroid cancer Aug 26, 2022
<ѻý>Nurture Over Nature: Environment Plays Greater Role in Teen Psychosisѻý> Genetics contributed less with more exposures to environmental risk factors, research suggests Aug 03, 2022
<ѻý>Rare Mutations Linked to Protection Against Liver Diseaseѻý> Carriers of CIDEB variants were at lower risk for liver disease of any cause, study shows Jul 27, 2022
<ѻý>Starch for Cancer Prevention? It's Not as Bananas as You Thinkѻý> Resistant starch may be protective against certain Lynch syndrome-related cancers Jul 25, 2022
<ѻý>'A New Hope' for Rare, Deadly Liver Diseaseѻý> Small fazirsiran trial sees fibrosis regressions for liver disease associated with AAT deficiency Jun 28, 2022
<ѻý>FDA Panel Backs Gene Tx for Degenerative Brain Disorder in Kidsѻý> Advisers unanimously recommend approval for pediatric ALD patients without HLA-matched donors Jun 10, 2022
<ѻý>T-Cell Receptor Gene Therapy Shows Promise in Pancreatic Cancerѻý> Novel treatment targeting KRAS G12D expression induces deep and durable response Jun 01, 2022
<ѻý>Do Pediatric Electrophysiologists Have It Harder?ѻý> Clinicians discuss the dilemmas and shortcomings in the field May 02, 2022
<ѻý>FDA Issues Warning on Prenatal Genetic Testsѻý> Relying on results could lead to inappropriate medical decisions, agency warns Apr 19, 2022
<ѻý>Gilbert Gottfried's Myotonic Dystrophyѻý> The genetic condition that claimed the life of the acclaimed comedian Apr 15, 2022
<ѻý>New Clue to Risk of Peritoneal Cancer After Prophylactic Salpingo-Oophorectomyѻý> Women with BRCA variant and serous tubal intraepithelial carcinoma had a 34-fold greater risk Apr 05, 2022
<ѻý>First Topical Therapy for Facial Angiofibroma Nabs FDA Approvalѻý> New alternative to dermabrasion, laser treatment, surgery Apr 04, 2022
<ѻý>The Evolving Role of the Medical Geneticistѻý> ACMG leadership on new opportunities for clinicians and the future of genetic medicine Mar 23, 2022
<ѻý>Experts Question Whether It's Okay to Test Embryos for Common Diseasesѻý> Researchers developed an algorithm to test for diseases such as diabetes, but is it ethical? Mar 21, 2022
<ѻý>Antisense Drug Slashed Hereditary Angioedema Attacks in Small Studyѻý> Donidalorsen also allowed longer intervals between treatments Mar 16, 2022
<ѻý>Prenatal Blood Test Info Often Misleads, Endangering Normal Pregnanciesѻý> Report blasts misleading language around non-invasive prenatal blood tests Mar 14, 2022
<ѻý>'Small Data' Goes a Long Way in Rare Disease Researchѻý> A close doctor-patient relationship can make all the difference Feb 28, 2022
<ѻý>Genetic Role of Alcohol-Cancer Link Strengthenedѻý> In study from China, two common variants associated with lower risk of cancer in men Feb 01, 2022
<ѻý>Gene Therapy Shows Clinical Benefit in Metachromatic Leukodystrophyѻý> Motor development, cognitive function preserved in early-onset patients Jan 24, 2022
<ѻý>The Real Reason Some Newborns Don't Get Screening for Deadly Diseasesѻý> Why do newborn screening tests vary so much from state to state? Dec 21, 2021
<ѻý>Highly Mutated Breast Cancer Responds to Immunotherapyѻý> Nivolumab-ipilimumab combination associated with durable response in small study Dec 09, 2021
<ѻý>Genetic Testing Benefits Children With Unexplained Epilepsyѻý> Findings can alter treatment, prognosis, overall care Dec 05, 2021
<ѻý>Preimplantation Genetic Testing Fails to Improve IVF Birth Ratesѻý> Testing not necessary for women with good chance of live birth, experts say Nov 24, 2021
<ѻý>FDA Panel Shows Little Love for Prader-Willi Drug Candidateѻý> All but one committee member said phase III evidence for carbetocin nasal spray was insufficient Nov 05, 2021
<ѻý>Ibrutinib Boosts Survival in Younger Patients With Certain DLBCL Subtypesѻý> New analysis "casts the PHOENIX trial in a new light," authors say Nov 04, 2021
<ѻý>Common Gene Variant May Predict Peritoneal Dialysis Outcomesѻý> Study also suggests that one dialysate type is better for carriers Oct 20, 2021
<ѻý>Gene Expression Test Improves IPF Diagnosisѻý> Quasi-real-world study indicates that physicians will likely find it helpful Oct 18, 2021