<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>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>Antisense Drug Slashed Hereditary Angioedema Attacks in Small Studyѻýҕl> Donidalorsen also allowed longer intervals between treatments Mar 16, 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
<ѻýҕl>Gene Therapy Shows Clinical Benefit in Metachromatic Leukodystrophyѻýҕl> Motor development, cognitive function preserved in early-onset patients Jan 24, 2022
<ѻýҕl>The Real Reason Some Newborns Don't Get Screening for Deadly Diseasesѻýҕl> Why do newborn screening tests vary so much from state to state? Dec 21, 2021
<ѻýҕl>FDA Panel Shows Little Love for Prader-Willi Drug Candidateѻýҕl> All but one committee member said phase III evidence for carbetocin nasal spray was insufficient Nov 05, 2021
<ѻýҕl>Ibrutinib Boosts Survival in Younger Patients With Certain DLBCL Subtypesѻýҕl> New analysis "casts the PHOENIX trial in a new light," authors say Nov 04, 2021
<ѻýҕl>Common Gene Variant May Predict Peritoneal Dialysis Outcomesѻýҕl> Study also suggests that one dialysate type is better for carriers Oct 20, 2021
<ѻýҕl>Gene Expression Test Improves IPF Diagnosisѻýҕl> Quasi-real-world study indicates that physicians will likely find it helpful Oct 18, 2021
<ѻýҕl>More Severe Congenital Heart Disease Tied to Lower IQ in Kidsѻýҕl> IQ reduced by 10 points compared with healthy peers Sep 24, 2021
<ѻýҕl>Liver Toxicity in Gene Therapy Trials Could Merit Stricter Entry Criteriaѻýҕl> FDA panel weighs in on lower vector doses and role of animal studies Sep 03, 2021
<ѻýҕl>A Familiar Drug Can Keep Early-Stage HCM at Bayѻýҕl> Treatment with valsartan most beneficial when started early? Aug 27, 2021
<ѻýҕl>Genetics, Socioeconomic Factors Each Tied to Diabetes and Obesityѻýҕl> People with highest risk genetics more than 15 times more likely to have T2D Jun 30, 2021