Remove Genetic Disease Remove Hormones Remove Life Science
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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.

Genetics 116
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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

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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.

Trials 98