Rare Mutations Linked to Protection Against Liver Disease Carriers of CIDEB variants were at lower risk for liver disease of any cause, study shows Jul 27, 2022
Starch for Cancer Prevention? It's Not as Bananas as You Think Resistant starch may be protective against certain Lynch syndrome-related cancers Jul 25, 2022
Familial Cancer Risk Knowledge Doesn't Spur Healthier Choices in Young People Maternal BRCA-positive status not tied to less tobacco or alcohol use in teens or young adults Jul 21, 2022
Does Pharmacogenomic Testing Improve Outcomes in Major Depressive Disorder? The evidence is unclear Jul 12, 2022
'A New Hope' for Rare, Deadly Liver Disease Small fazirsiran trial sees fibrosis regressions for liver disease associated with AAT deficiency Jun 28, 2022
Beta-Thalassemia Gene Tx Gets Ringing Endorsement From FDA Advisers "Outstanding" efficacy, "life-changing" for transfusion-dependent patients, says panelist Jun 10, 2022
FDA Panel Backs Gene Tx for Degenerative Brain Disorder in Kids Advisers unanimously recommend approval for pediatric ALD patients without HLA-matched donors Jun 10, 2022
T-Cell Receptor Gene Therapy Shows Promise in Pancreatic Cancer Novel treatment targeting KRAS G12D expression induces deep and durable response Jun 01, 2022
How to Inactivate Pathogens; Treat Long COVID With Vaccine? Also in TTHealthWatch: unintended consequences of a cancer treatment May 28, 2022 podcast
FDA's IT Systems Are '20 Years Behind' Industry Standard, Official Says Fixing the problem will require "a fair amount of investment," says CBER chief Peter Marks May 11, 2022
Do Pediatric Electrophysiologists Have It Harder? Clinicians discuss the dilemmas and shortcomings in the field May 02, 2022
FDA Issues Warning on Prenatal Genetic Tests Relying on results could lead to inappropriate medical decisions, agency warns Apr 19, 2022
Good News About Cognition for Those Predisposed to High Cholesterol How do statins factor between familial hypercholesterolemia and dementia risk? Apr 19, 2022
Gilbert Gottfried's Myotonic Dystrophy The genetic condition that claimed the life of the acclaimed comedian Apr 15, 2022
First-in-Class Drug Thwarts Hemolytic Anemia in Adults With PK Deficiency Mitapivat improved outcomes in "substantial minority" of patients with rare hereditary condition Apr 14, 2022
New Clue to Risk of Peritoneal Cancer After Prophylactic Salpingo-Oophorectomy Women with BRCA variant and serous tubal intraepithelial carcinoma had a 34-fold greater risk Apr 05, 2022
First Topical Therapy for Facial Angiofibroma Nabs FDA Approval New alternative to dermabrasion, laser treatment, surgery Apr 04, 2022
Herd Immunity Out of Reach? House Caps Insulin $$; First Full Human Genome Map A daily roundup of news on COVID-19 and the rest of medicine Apr 01, 2022
The Evolving Role of the Medical Geneticist ACMG leadership on new opportunities for clinicians and the future of genetic medicine Mar 23, 2022
Experts Question Whether It's Okay to Test Embryos for Common Diseases Researchers developed an algorithm to test for diseases such as diabetes, but is it ethical? Mar 21, 2022
COVID Secondary Brain Changes; Screening Normal-Weight People for Eating Disorders Also in TTHealthWatch: gene therapy for hemophilia A Mar 19, 2022 podcast
How Is the U.S. Watching for the Next COVID Variant? While sequencing efforts are better than before, experts say there's a long way to go Mar 18, 2022
Antisense Drug Slashed Hereditary Angioedema Attacks in Small Study Donidalorsen also allowed longer intervals between treatments Mar 16, 2022
Gene Therapy Impresses in Severe Hemophilia A Valoctocogene roxaparvovec significantly increased factor VIII activity, reduced bleeding Mar 16, 2022
Prenatal Blood Test Info Often Misleads, Endangering Normal Pregnancies Report blasts misleading language around non-invasive prenatal blood tests Mar 14, 2022
Kidney Disease Gene Risk Info Aids Prevention in Primary Care Trial showed benefits in hypertensive patients of African descent Mar 04, 2022
'Small Data' Goes a Long Way in Rare Disease Research A close doctor-patient relationship can make all the difference Feb 28, 2022
Genetic Role of Alcohol-Cancer Link Strengthened In study from China, two common variants associated with lower risk of cancer in men Feb 01, 2022
DCM Screening Often Turns Up Familial Cases Risk 89% higher for families of Black patients with dilated cardiomyopathy Feb 01, 2022
Gene Therapy Shows Clinical Benefit in Metachromatic Leukodystrophy Motor development, cognitive function preserved in early-onset patients Jan 24, 2022
No, 'Deltacron' Is Probably Not Real Reports of a 'recombinant' variant likely just hinge on lab contamination Jan 10, 2022
The Real Reason Some Newborns Don't Get Screening for Deadly Diseases Why do newborn screening tests vary so much from state to state? Dec 21, 2021
Highly Mutated Breast Cancer Responds to Immunotherapy Nivolumab-ipilimumab combination associated with durable response in small study Dec 09, 2021
Genetic Testing Benefits Children With Unexplained Epilepsy Findings can alter treatment, prognosis, overall care Dec 05, 2021
Preimplantation Genetic Testing Fails to Improve IVF Birth Rates Testing not necessary for women with good chance of live birth, experts say Nov 24, 2021
Hemophilia Gene Therapy Looks Like a Winner Most patients do well in 3-year phase I/II study Nov 17, 2021
Mind-Heart Health Connection; 100,000 Genome Project Also this week in TTHealthWatch: Seropositivity, COVID-19, and persistent symptoms Nov 13, 2021 podcast
FDA Panel Shows Little Love for Prader-Willi Drug Candidate All but one committee member said phase III evidence for carbetocin nasal spray was insufficient Nov 05, 2021
Ibrutinib Boosts Survival in Younger Patients With Certain DLBCL Subtypes New analysis "casts the PHOENIX trial in a new light," authors say Nov 04, 2021