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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. A base-editing approach could also likely be a one-time treatment for all prion disease patients regardless of the genetic mutation causing their disease.

Disease 144
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Chemotherapy-free treatment for patients with blood cancer

Drug Target Review

An important research area at AbbVie involves blood cancers, where our ultimate goal is to transform standards of care across these diseases and advance a dynamic cancer research and treatment pipeline. 2 However, treatment options were still limited and often came with severe side effects.

Treatment 105
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Fewer than half of new drugs add substantial therapeutic value over existing treatments

SCIENMAG: Medicine & Health

But a study published by The BMJ today finds that less than half of approved first indications for new drugs in the US and Europe between 2011 and 2020 add substantial therapeutic value over existing treatments […]

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Patient Perspective: Jamie Troil Goldfarb shares her clinical trial experience [video]

Antidote

Not only will patients be able to gain access to potential new treatments, but they will also be directly contributing to research that can save lives — something that Jamie Troil Goldfarb has experienced firsthand. Jamie received a diagnosis of stage IV melanoma in 2011, only 11 weeks after giving birth to her child.

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Study finds acute alcohol poisoning is a significant component of alcohol-related mortality

National Drug & Alcohol Research Centre Blog

The study, published in Drug and Alcohol Review, examined deaths from acute alcohol poisoning in Australia, 2011–2022. Are these inexperienced drinkers with a low tolerance or seasoned users with long histories of alcohol problems, perhaps receiving treatment for alcohol dependence? says lead author Professor Shane Darke.

Disease 110
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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it. A Long and Winding Diagnostic Road The story begins in 2011, with Alessandra Biffi, MD, head of the clinical trial for MLD at the San Raffaele Telethon Institute for Gene Therapy in Milan.

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Sygnature Discovery Unlocks Life-Changing Treatment Options with New Human Disease Modeling Platform

Sygnature Discovery

Sygnature Discovery has unveiled its humanized neuroinflammation in vitro platform, SCANME (SCreening for Anti-Neuroinflammation Molecular Entities), designed to scale up human disease modeling and push the boundaries of treatment for life-changing neuroinflammation related conditions such as Alzheimer’s disease.

Disease 52