<ѻýҕl>Rare Disease Non-Profit Shows Drug Development Savvyѻýҕl> FAST sold a drug candidate to seed other treatment strategies for Angelman syndrome Feb 15, 2023
<ѻýҕl>Does the Environment Override Genes for People at Risk of Stroke?ѻýҕl> Large study detects interaction between genetic susceptibility and neighborhood deprivation Feb 08, 2023
<ѻýҕl>Inflammatory VEXAS Syndrome May Not Be So Uncommonѻýҕl> Is new disease being missed by physicians? Jan 24, 2023
<ѻýҕl>We're Underutilizing Genetic Testing for Colorectal Cancerѻýҕl> Universal germline testing should be part of the standard of cancer care Jan 20, 2023
<ѻýҕl>Genomic Assay May Pinpoint Who Can Skip RT After Breast-Conserving Surgeryѻýҕl> Patients categorized as low risk with 16-gene signature showed no significant benefit with RT Jan 06, 2023
<ѻýҕl>Dementia Accompanying Cardiometabolic Disease: Does It Come Down to Genetics?ѻýҕl> Twin study suggests special monitoring in some individuals Dec 29, 2022
<ѻýҕl>Families Push Research Forward in Rare Diseasesѻýҕl> The field has a unique funding model. While fruitful, some question if it needs to change. Dec 26, 2022
<ѻýҕl>COVID Vaccines Made mRNA a Household Name. How Can It Help in Rare Diseases?ѻýҕl> A handful of companies have mRNA therapies in human trials for rare diseases Dec 26, 2022
<ѻýҕl>Early Win for Gene Therapy in Rare Form of SCIDѻýҕl> 10 infants were able to make their own T cells; safety good so far Dec 21, 2022
<ѻýҕl>Topical Gene Therapy Heals Blistering Skin Disease Lesionsѻýҕl> Randomized trial shows "dramatic" benefits in rare genetic disease Dec 14, 2022
<ѻýҕl>Assay Predicts Breast Cancer Patients Who Will Benefit From Ovarian Suppressionѻýҕl> Low score suggests improved outcomes with ovarian suppression plus adjuvant endocrine therapy Dec 07, 2022
<ѻýҕl>Decoding Cancerѻýҕl> A conversation with Harold Varmus, MD, who received the Nobel Prize in Physiology or Medicine in 1989 Dec 06, 2022 podcast
<ѻýҕl>Chris Hemsworth's APOE4 Alzheimer's Geneѻýҕl> The Thor actor recently learned about his genetic predisposition Dec 01, 2022
<ѻýҕl>FDA OKs First Gene Therapy for Hemophilia Bѻýҕl> Hemgenix reduced rate of annual bleeds, eliminated need for factor IX prophylaxis therapy Nov 22, 2022
<ѻýҕl>Early Cardiac Amyloidosis Still Bad News Without Treatmentѻýҕl> Observational study supports treating even asymptomatic patients Nov 16, 2022
<ѻýҕl>CRISPR Shows Promise for Hereditary Angioedema in First-in-Human Trialѻýҕl> No safety concerns reported and swelling attacks declined Nov 15, 2022
<ѻýҕl>Promising Results in First Test of In Utero Enzyme-Replacement Therapyѻýҕl> Treatment was safe, effective in one baby with infantile-onset Pompe disease Nov 09, 2022
<ѻýҕl>My Risk Revealed: A Confirmed BRCA2 Mutationѻýҕl> My dad's cancer diagnosis offered me invaluable information Oct 30, 2022
<ѻýҕl>Texas Says DNA Kits for Schoolchildren Not Tied to Uvalde Shootingѻýҕl> State education agency says effort was designed to help law enforcement find missing kids Oct 27, 2022
<ѻýҕl>Gene Therapy for Degenerative Brain Disorder Wins FDA Nodѻýҕl> $3 million price tag for one-time treatment Sep 19, 2022
<ѻýҕl>Blood Type Tied to Early-Onset Stroke Riskѻýҕl> Findings point to prothrombotic mechanisms in younger stroke patients Sep 01, 2022
<ѻýҕl>Pralsetinib Shows Promise in Diverse RET Fusion-Positive Cancersѻýҕl> An ORR of 57% supports results previously observed in NSCLC and thyroid cancer Aug 26, 2022
<ѻýҕl>Nurture Over Nature: Environment Plays Greater Role in Teen Psychosisѻýҕl> Genetics contributed less with more exposures to environmental risk factors, research suggests Aug 03, 2022
<ѻýҕl>Rare Mutations Linked to Protection Against Liver Diseaseѻýҕl> Carriers of CIDEB variants were at lower risk for liver disease of any cause, study shows Jul 27, 2022
<ѻýҕl>Starch for Cancer Prevention? It's Not as Bananas as You Thinkѻýҕl> Resistant starch may be protective against certain Lynch syndrome-related cancers Jul 25, 2022
<ѻýҕl>'A New Hope' for Rare, Deadly Liver Diseaseѻýҕl> Small fazirsiran trial sees fibrosis regressions for liver disease associated with AAT deficiency Jun 28, 2022
<ѻýҕl>FDA Panel Backs Gene Tx for Degenerative Brain Disorder in Kidsѻýҕl> Advisers unanimously recommend approval for pediatric ALD patients without HLA-matched donors Jun 10, 2022
<ѻýҕl>T-Cell Receptor Gene Therapy Shows Promise in Pancreatic Cancerѻýҕl> Novel treatment targeting KRAS G12D expression induces deep and durable response Jun 01, 2022
<ѻýҕl>Do Pediatric Electrophysiologists Have It Harder?ѻýҕl> Clinicians discuss the dilemmas and shortcomings in the field May 02, 2022
<ѻýҕl>FDA Issues Warning on Prenatal Genetic Testsѻýҕl> Relying on results could lead to inappropriate medical decisions, agency warns Apr 19, 2022
<ѻýҕl>Gilbert Gottfried's Myotonic Dystrophyѻýҕl> The genetic condition that claimed the life of the acclaimed comedian Apr 15, 2022
<ѻýҕl>New Clue to Risk of Peritoneal Cancer After Prophylactic Salpingo-Oophorectomyѻýҕl> Women with BRCA variant and serous tubal intraepithelial carcinoma had a 34-fold greater risk Apr 05, 2022
<ѻýҕl>First Topical Therapy for Facial Angiofibroma Nabs FDA Approvalѻýҕl> New alternative to dermabrasion, laser treatment, surgery Apr 04, 2022
<ѻýҕl>The Evolving Role of the Medical Geneticistѻýҕl> ACMG leadership on new opportunities for clinicians and the future of genetic medicine Mar 23, 2022
<ѻýҕl>Experts Question Whether It's Okay to Test Embryos for Common Diseasesѻýҕl> Researchers developed an algorithm to test for diseases such as diabetes, but is it ethical? Mar 21, 2022
<ѻýҕl>Antisense Drug Slashed Hereditary Angioedema Attacks in Small Studyѻýҕl> Donidalorsen also allowed longer intervals between treatments Mar 16, 2022
<ѻýҕl>Prenatal Blood Test Info Often Misleads, Endangering Normal Pregnanciesѻýҕl> Report blasts misleading language around non-invasive prenatal blood tests Mar 14, 2022
<ѻýҕl>'Small Data' Goes a Long Way in Rare Disease Researchѻýҕl> A close doctor-patient relationship can make all the difference Feb 28, 2022
<ѻýҕl>Genetic Role of Alcohol-Cancer Link Strengthenedѻýҕl> In study from China, two common variants associated with lower risk of cancer in men Feb 01, 2022