<乌鸦传媒>FDA 'Leans In' to Accelerated Approval for Rare Disease Drugs CBER director says "moment is tender for gene therapy," sees opportunity to "salvage" treatments May 19, 2023
<乌鸦传媒>ASCO Backs Routine ESR1 Testing for Certain Breast Cancers Activity of elacestrant in EMERALD trial "provides strong signal" for guideline update May 18, 2023
<乌鸦传媒>Alzheimer's Progression Delayed by Rare Genetic Variant Finding challenges previous assumptions about disease May 15, 2023
<乌鸦传媒>B-Cell Stimulating Factors Tied to Lymphoma in Sjogren's Findings may bolster interest in BTK inhibitors for autoimmune diseases May 02, 2023
<乌鸦传媒>FDA Extends Trikafta Approval to Even Younger Cystic Fibrosis Patients Patients ages 2 to 5 years now eligible for the triple therapy Apr 27, 2023
<乌鸦传媒>Genomics Add Insight Into Worse Colorectal Cancer Outcomes in Black Patients Fewer actionable mutations, less likely to meet genomic criteria for immunotherapy Apr 19, 2023
<乌鸦传媒>Many With Cancer Predisposition Diseases Unaware of Genetic Status Moreover, a significant proportion do not qualify for genetic screening under current guidelines Apr 19, 2023
<乌鸦传媒>Oral Drug Improves Sunlight Tolerance in Genetic Photosensitivity Disorders Dersimelagon significantly extended time to sun-related prodromal symptoms Apr 12, 2023
<乌鸦传媒>Genomics Can Help Diagnose Rare Pediatric Diseases Large study found genomic analysis delivered answers for 41% of kids with undiagnosed disease Apr 12, 2023
<乌鸦传媒>NIH's Genome Chief Says Genomics Is Evolving and Docs Need More Education on It National Human Genome Research Institute Director Eric Green sits down with 乌鸦传媒 Apr 07, 2023
<乌鸦传媒>A 'Double Whammy' for Gastric Cancer Risk H. pylori infection and certain pathogenic variants linked with large excess risk Mar 29, 2023
<乌鸦传媒>Common Enzyme Deficiency Linked With COVID Severity Certain groups of men with G6PD deficiency had higher odds of severe illness Mar 29, 2023
<乌鸦传媒>FDA Greenlights First Drug for Rare Immunodeficiency Disease Activated PI3K-delta syndrome may have a new standard of care Mar 27, 2023
<乌鸦传媒>Investigational ALS Drug May Have Clinical Benefit, FDA Staff Says Neurofilament light will be evaluated as a surrogate endpoint in tofersen review Mar 20, 2023
<乌鸦传媒>Fetus Removed From Brain of 1-Year-Old Girl Intracranial fetus-in-fetu identified in child with motor delay Mar 09, 2023
<乌鸦传媒>KRAS Mutations in Metastatic CRC Predict Survival Benefit of Chemo Combo Trifluridine/tipiracil fails to improve survival in patients with KRAS G12-mutant tumors Mar 06, 2023
<乌鸦传媒>Expanding ICD Codes May Aid Rare Disease Patients, Research NCATS explores how best to use diagnostic codes to move field forward Mar 02, 2023
<乌鸦传媒>First Treatment Approved for Rare Neurodegenerative Disorder Omaveloxolone gets FDA nod for Friedreich's ataxia Mar 01, 2023
<乌鸦传媒>FDA OKs First Therapy for Alpha-Mannosidosis Velmanase alfa approved for treating non-CNS manifestations of progressive, inherited disease Feb 17, 2023
<乌鸦传媒>Rare Disease Non-Profit Shows Drug Development Savvy FAST sold a drug candidate to seed other treatment strategies for Angelman syndrome Feb 15, 2023
<乌鸦传媒>Does the Environment Override Genes for People at Risk of Stroke? Large study detects interaction between genetic susceptibility and neighborhood deprivation Feb 08, 2023
<乌鸦传媒>Inflammatory VEXAS Syndrome May Not Be So Uncommon Is new disease being missed by physicians? Jan 24, 2023
<乌鸦传媒>We're Underutilizing Genetic Testing for Colorectal Cancer Universal germline testing should be part of the standard of cancer care Jan 20, 2023
<乌鸦传媒>Genomic Assay May Pinpoint Who Can Skip RT After Breast-Conserving Surgery Patients categorized as low risk with 16-gene signature showed no significant benefit with RT Jan 06, 2023
<乌鸦传媒>Dementia Accompanying Cardiometabolic Disease: Does It Come Down to Genetics? Twin study suggests special monitoring in some individuals Dec 29, 2022
<乌鸦传媒>Families Push Research Forward in Rare Diseases The field has a unique funding model. While fruitful, some question if it needs to change. Dec 26, 2022
<乌鸦传媒>COVID Vaccines Made mRNA a Household Name. How Can It Help in Rare Diseases? A handful of companies have mRNA therapies in human trials for rare diseases Dec 26, 2022
<乌鸦传媒>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
<乌鸦传媒>Topical Gene Therapy Heals Blistering Skin Disease Lesions Randomized trial shows "dramatic" benefits in rare genetic disease Dec 14, 2022
<乌鸦传媒>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
<乌鸦传媒>Decoding Cancer A conversation with Harold Varmus, MD, who received the Nobel Prize in Physiology or Medicine in 1989 Dec 06, 2022 podcast
<乌鸦传媒>Chris Hemsworth's APOE4 Alzheimer's Gene The Thor actor recently learned about his genetic predisposition Dec 01, 2022
<乌鸦传媒>FDA OKs First Gene Therapy for Hemophilia B Hemgenix reduced rate of annual bleeds, eliminated need for factor IX prophylaxis therapy Nov 22, 2022
<乌鸦传媒>Early Cardiac Amyloidosis Still Bad News Without Treatment Observational study supports treating even asymptomatic patients Nov 16, 2022
<乌鸦传媒>CRISPR Shows Promise for Hereditary Angioedema in First-in-Human Trial No safety concerns reported and swelling attacks declined Nov 15, 2022
<乌鸦传媒>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
<乌鸦传媒>My Risk Revealed: A Confirmed BRCA2 Mutation My dad's cancer diagnosis offered me invaluable information Oct 30, 2022
<乌鸦传媒>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
<乌鸦传媒>Gene Therapy for Degenerative Brain Disorder Wins FDA Nod $3 million price tag for one-time treatment Sep 19, 2022
<乌鸦传媒>Blood Type Tied to Early-Onset Stroke Risk Findings point to prothrombotic mechanisms in younger stroke patients Sep 01, 2022
