Early Win for Gene Therapy in Rare Form of SCID 10 infants were able to make their own T cells; safety good so far Dec 21, 2022
Topical Gene Therapy Heals Blistering Skin Disease Lesions Randomized trial shows "dramatic" benefits in rare genetic disease Dec 14, 2022
Assay Predicts Breast Cancer Patients Who Will Benefit From Ovarian Suppression Low score suggests improved outcomes with ovarian suppression plus adjuvant endocrine therapy Dec 07, 2022
Decoding Cancer A conversation with Harold Varmus, MD, who received the Nobel Prize in Physiology or Medicine in 1989 Dec 06, 2022 podcast
Chris Hemsworth's APOE4 Alzheimer's Gene The Thor actor recently learned about his genetic predisposition Dec 01, 2022
FDA OKs First Gene Therapy for Hemophilia B Hemgenix reduced rate of annual bleeds, eliminated need for factor IX prophylaxis therapy Nov 22, 2022
Early Cardiac Amyloidosis Still Bad News Without Treatment Observational study supports treating even asymptomatic patients Nov 16, 2022
CRISPR Shows Promise for Hereditary Angioedema in First-in-Human Trial No safety concerns reported and swelling attacks declined Nov 15, 2022
Promising Results in First Test of In Utero Enzyme-Replacement Therapy Treatment was safe, effective in one baby with infantile-onset Pompe disease Nov 09, 2022
My Risk Revealed: A Confirmed BRCA2 Mutation My dad's cancer diagnosis offered me invaluable information Oct 30, 2022
Texas Says DNA Kits for Schoolchildren Not Tied to Uvalde Shooting State education agency says effort was designed to help law enforcement find missing kids Oct 27, 2022
Gene Therapy for Degenerative Brain Disorder Wins FDA Nod $3 million price tag for one-time treatment Sep 19, 2022
Blood Type Tied to Early-Onset Stroke Risk Findings point to prothrombotic mechanisms in younger stroke patients Sep 01, 2022
Pralsetinib Shows Promise in Diverse RET Fusion-Positive Cancers An ORR of 57% supports results previously observed in NSCLC and thyroid cancer Aug 26, 2022
Nurture Over Nature: Environment Plays Greater Role in Teen Psychosis Genetics contributed less with more exposures to environmental risk factors, research suggests Aug 03, 2022
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
'A New Hope' for Rare, Deadly Liver Disease Small fazirsiran trial sees fibrosis regressions for liver disease associated with AAT deficiency Jun 28, 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
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
Gilbert Gottfried's Myotonic Dystrophy The genetic condition that claimed the life of the acclaimed comedian Apr 15, 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
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
Antisense Drug Slashed Hereditary Angioedema Attacks in Small Study Donidalorsen also allowed longer intervals between treatments 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
'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
Gene Therapy Shows Clinical Benefit in Metachromatic Leukodystrophy Motor development, cognitive function preserved in early-onset patients Jan 24, 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
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
Common Gene Variant May Predict Peritoneal Dialysis Outcomes Study also suggests that one dialysate type is better for carriers Oct 20, 2021
