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The FIND FH model can successfully identify individuals with familial hypercholesterolemia from a scan of large

AI Model Helps ID Those at Risk for Familial Hypercholesterolemia

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Model may identify more who are at risk while substantially reducing the number of people screened
Initiation of statin therapy during childhood in patients with familial hypercholesterolemia slows the progression of carotid intima-media thickness and reduces the risk for cardiovascular disease over 20 years

Statins in Childhood Aid Familial Hypercholesteremia Outcomes

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Patients show slowed progression of carotid intima-media thickness, lower risk for cardiovascular disease
A triplet combination of therapies (encorafenib

Triplet Tx Ups Survival in BRAF V600E-Mutated Colorectal Cancer

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Overall survival longer for encorafenib, cetuximab, and binimetinib combo versus standard therapy
The first attempt to use the gene-editing tool CRISPR to cure HIV infection was unsuccessful but the approach does show promise.

Scientists Use Gene-Editing Tool CRISPR in Attempt to Cure HIV

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Attempt failed, but tool appears precise and safe and holds promise
Use of a response- and biology-based treatment algorithm for intermediate-risk neuroblastoma is associated with excellent survival and reduces treatment for some patients

Algorithm Based on Response, Biology Guides Neuroblastoma Tx

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Survival excellent with treatment algorithm for children with intermediate-risk neuroblastoma
The U.S. Preventive Services Task Force recommends that women who are at increased risk for BRCA1/2 mutations should undergo genetic counseling

USPSTF Recommends Genetic Counseling for High BRCA Risk

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But counseling not recommended for women without personal, family history, ancestry associated with BRCA mutations
A considerable proportion of families diagnosed at an autosomal dominant tubulointerstitial kidney disease specialist center are the result of direct family referral from the internet

Internet Self-Referrals Aid in Diagnosis of Rare Inherited Kidney Disease

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Twenty-four percent of direct family referrals to specialist center had positive genetic testing
First-degree relatives of patients with hematological malignancies have increased relative risks for the same tumor types and some different hematological malignancies

Hematological Malignancy Risk Up for First-Degree Relatives

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Increased familial relative risks for the same tumor type for most hematological malignancies
A U.S. clinical trial of CRISPR gene-editing to treat people with sickle cell disease is underway.

Trial Tests CRISPR Gene-Editing to Treat Sickle Cell Disease

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Study seeks to recruit up to 45 adults with severe sickle cell disease
In a U.S. first

First U.S. Trial Using CRISPR Within the Body Set to Begin

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Researchers will attempt to give patients with Leber congenital amaurosis a healthy version of the gene they lack