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In a first, children with rare genetic diseases get mitochondrial transplants from their mothers

STAT News

They also metabolize cholesterol and synthesize hormones and neurotransmitters. And over the next 2 billion or so years, that bacteria evolved to be the mitochondria that power nearly every cell in the human body. These capsule-shaped organelles don’t just turn oxygen and nutrients into chemical energy.

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Researchers show novel device improves blood sugar control in hyperinsulinism patients whose pancreas has been removed

Scienmag

Philadelphia, September 13, 2021—Researchers at Children’s Hospital of Philadelphia (CHOP) have demonstrated that an experimental device can improve blood sugar control in patients who developed diabetes after their pancreas was removed to treat their hyperinsulinism, a genetic disease in which the pancreas produces too much insulin.

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Hypoxia-Inducible Factor-2 Alpha (HIF-2?) Inhibitor WELIREG™ (belzutifan) for the Treatment of Patients With Certain Types of Von Hippel-Lindau (VHL) Disease-Associated Tumors

The Pharma Data

Advise patients of these risks and the need for effective non-hormonal contraception. WELIREG can render some hormonal contraceptives ineffective. About Von Hippel-Lindau Disease. This is a rare genetic disease with an estimated incidence of 10,000 people in the U.S.

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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

For the following 15 rare diseases, all prevalence (approximate), symptom, etiology and treatment information was taken from the National Organization for Rare Disorders’ (NORD) Rare Disease Database , unless a link to another source is provided. Cushing’s Disease. Acromegaly. Prevalence: 50 to 70 people per 1 million.

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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. They can provide continuous real-time monitoring of metabolites, bacteria, hormones, glucose and other elements in these fluids. RNA Therapeutics. The deal is worth $1.5 Prior to this, Lilly struck an agreement worth $1.25