乌鸦传媒

Genetics

<乌鸦传媒 class='mpt-top_cat_title_description'>News, opinions and meeting coverage in genetics.

A computer rendering of single strands of RNA.

<乌鸦传媒>First Cases of New Neurology Syndrome Identified

Long non-coding RNA implicated in human disease

AAO over a photo of McCormick Place in Chicago, IL.

<乌鸦传媒>Evidence of Durable Vision Improvement With Gene Therapy for Retinitis Pigmentosa

Significant improvement beyond 100 weeks after single injection of mutation-agnostic MCO-010

AANEM over a photo of Savannah Convention Center in Savannah, GA.

<乌鸦传媒>Gene Therapy Details Revealed in Duchenne Trial

Differences between delandistrogene moxeparvovec and placebo emerge in secondary outcomes

A computer rendering of a transparent body with the large intestine highlighted.

<乌鸦传媒>Allele May Heighten Risk of Severe Ulcerative Colitis

Finding on HLA-DRB1*01:03 supports need for more and earlier genetic studies

FDA ADCOMM elamipretide hydrochloride injection over a computer rendering of mitochondria.

<乌鸦传媒>Elamipretide for Barth Syndrome Survives FDA Advisory Vote

Panelists acknowledge difficulty generating stronger data in ultra-rare disease

A computer rendering of stem cells and a strand of DNA.

<乌鸦传媒>Gene Therapy for Rare Brain Disease Effective, but Comes With Blood Cancer Risk

10% of patients in trials leading to eli-cel's approval developed MDS or AML

FDA ADCOMM elamipretide hydrochloride injection over a computer rendering of mitochondria.

<乌鸦传媒>FDA Reviewers Cast Doubt on Drug Candidate for Barth Syndrome

Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease

A photo of various types of hormonal contraception.

<乌鸦传媒>Hormonal Contraception Linked to Increased Breast Cancer Risk in BRCA1 Carriers

Higher risk for ever use, increasing duration of use but no increase in BRCA2 carriers

FDA APPROVED levacetylleucine (Aqneursa) over a computer rendering of mutating DNA.

<乌鸦传媒>Second Drug OK'd for Ultra-Rare Neurodegenerative Disease

FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C

FDA APPROVED arimoclomol (Miplyffa) over a computer rendering of a strand of DNA.

<乌鸦传媒>First Drug Approved for Niemann-Pick Disease Type C

Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder

A photo of an elderly patient receiving an intravenous drip while using a ventilator in Beijing.

<乌鸦传媒>New Genetic Analysis of Animals in Wuhan Market in 2019 May Help Find COVID's Origin

Findings "shift the dial more in favor of an animal origin," said one expert

Morning Break over illustration of a syringe, Covid virus, and DNA helix over a photo of green vegetation.

<乌鸦传媒>RFK Jr. Helping Trump Pick CDC Chief; Excess COVID Death Lingers; Turmoil at 23andMe

Health news and commentary gathered by 乌鸦传媒 staff

ESC over a photo of ExCeL London conference and exhibition center.

<乌鸦传媒>Novel Drug Reduced Triglycerides, Risk of Pancreatitis in Persistent Chylomicronemia

Benefits seen in patients with and without a genetic diagnosis

A computer rendering of red blood cells

<乌鸦传媒>A Miss for Steroid-Toting Red Blood Cells in Ataxia Telangiectasia

Phase III trial falls short of efficacy target, but positive signal emerges in younger kids

FDA ADCOMM arimoclomol over a computer rendering of a strand of DNA.

<乌鸦传媒>FDA Advisors Mostly Convinced Arimoclomol Works for Rare Genetic Disorder

If approved, this would be the first drug for Niemann-Pick disease type C

FDA ADCOMM arimoclomol over a computer rendering of a strand of DNA.

<乌鸦传媒>FDA Panel to Consider Arimoclomol for Rare Genetic Disorder

This is the second time the drug has been submitted to the FDA for Niemann-Pick disease type C

A computer rendered strand of mRNA.

<乌鸦传媒>Gene-Expression Profiling Can Help Improve Outcomes in Cancers of Unknown Primary

In randomized study, guided site-specific therapy improved PFS versus empirical chemotherapy

A computer rendering of an artery, capillary, and arteriole.

<乌鸦传媒>Sotorasib Promising for KRAS鈥揜elated Vascular Malformations

Two patients had rapid symptom improvement and reductions in malformation volume

ASRS over a photo of Stockholmsm盲ssan Convention Center in Stockholm, Sweden.

<乌鸦传媒>Gene Therapies for Neovascular AMD Continue to Show Promise

Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy

Morning Break over illustration of a syringe, Covid virus, and DNA helix over a photo of green vegetation.

<乌鸦传媒>Trump's Former Doc Weighs In; 23andMe and Alzheimer's Risk; What's 'Teflon Flu'?

Health news and commentary gathered by 乌鸦传媒 staff

A photo of the East London skyline under a layer of smog.

<乌鸦传媒>Psoriasis Joins List of Diseases Tied to Air Pollution

Poor air quality adds significantly to risk from genetic susceptibility factors

A photo of smog over London.

<乌鸦传媒>Genes, Air Pollution Combine to Raise Lupus Risk

British data suggest how environmental and genetic factors add together

A computer rendering of an oligonucleotide aptamer attacking a cancer cell.

<乌鸦传媒>Can Oligonucleotide Infusions Really Fix Lyme, Cancer, and Other Diseases?

U.S. clinics market oligonucleotide therapy for a wide range of conditions, without much evidence

A photo of people on the beach in Tel Aviv, Israel.

<乌鸦传媒>Adolescent Obesity Tied to Parents' BMI at the Same Age

Moderate association between parental and offspring BMI at age 17

FDA APPROVED crovalimab (Piasky) over a computer rendering of blood flowing inside a vessel.

<乌鸦传媒>Subcutaneous C5 Inhibitor Approved for PNH

Longer-acting crovalimab designed for more sustained effect and less treatment burden

A computer rendering of a meningioma.

<乌鸦传媒>ALK Inhibitor Shows Promise in Rare, Progressive Tumor Types

Phase II study found brigatinib exceeded historical control by shrinking 10% of targeted tumors

A photo of Robin Alderman and her son Camden Alderman.

<乌鸦传媒>Gene Therapy May Cure More Rare Diseases, but Drugmakers Have Few Incentives

Profitability, production cost, and low number of patients are barriers

A computer rendering of amyloid plaques on a nerve cell

<乌鸦传媒>Alzheimer's Onset Delayed by Rare Mutation

Just one copy of the Christchurch variant conferred protection

computer rendering of amyloid plaques amongst neurons and neurofibrillary tangles inside neurons

<乌鸦传媒>Alzheimer's Risk Higher If Mom Had Memory Problems

Maternal history of memory impairment tied to higher amyloid burden

A microscope image of the 46 human chromosomes

<乌鸦传媒>A Single, Surprising Gene Behind a Disorder That Causes Intellectual Disability

Researchers estimate the disorder may affect as many as one in 20,000 young people

A photo of a man鈥檚 lip swollen from hereditary angioedema

<乌鸦传媒>Hereditary Angioedema Attacks Reduced With Donidalorsen Prophylaxis

Investigational treatment passes muster in phase III trial

A computer rendering of amyloid plaques amongst neurons and neurofibrillary tangles inside neurons.

<乌鸦传媒>Lifestyle-Dementia Links Persist Regardless of Risk Genes, French Study Shows

Dementia prevention efforts may be beneficial despite genetic susceptibility

A computer rendering of in vitro fertilization.

<乌鸦传媒>Fertility Treatment Safe for Breast Cancer Survivors With BRCA Mutations

No significant increased risk of recurrence or pregnancy complications

A computer rendering of rod and cone photoreceptors

<乌鸦传媒>Gene Editing Improves Vision in Rare and Devastating Retinal Disorder

Therapy improved best corrected visual acuity in four of 14 patients, and others benefited too

A computer rendering of amyloid plaques on a nerve cell.

<乌鸦传媒>Alzheimer's Variant May Cause Distinct Form of the Disease

Almost all APOE4 homozygotes develop Alzheimer's disease

A photo of a box of Adzynma

<乌鸦传媒>Impact of Recombinant Replacement Therapy Detailed in Genetic Clotting Disorder

Trial that led to FDA approval shows a big difference in replacement levels versus usual care

A computer rendering of a blood vessel narrowed by atherosclerosis

<乌鸦传媒>Age-Related Somatic Mutations Implicated in Cardiovascular Death

Clonal hematopoiesis a red flag for people with asymptomatic carotid atherosclerosis

FDA APPROVED mavorixafor (Xolremdi) over a photo of warts on a woman鈥檚 hand.

<乌鸦传媒>FDA Approves First Drug for WHIM Syndrome

CXCR4 antagonist mavorixafor effectively boosts neutrophils and lymphocytes to reduce infections

Stylized illustration of strands of human DNA.

<乌鸦传媒>Genetics Studies Have a Diversity Problem That Researchers Struggle to Fix

Most data based on DNA from white people, but small differences can have big health outcomes

A computer rendering of a motor neuron connecting to a muscle fiber.

<乌鸦传媒>Newborn Screening for SMA Appears to Offer Functional Benefits

German cohort study suggests earlier initiation of treatment benefits sitting, walking

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