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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog

These have included clinical trials for rare diseases across metabolic (n=5), oncology (n=5), cardiology (n=3), dermatology (n=3), pulmonary (n=3), hematology (n=2), hepatology (n=2), ocular (n=2), transplant (n=1), gastrointestinal (n=1), immunology (n=1), infectious (n=1), neurology (n=1), orthopedic (n=1), and toxicology (n=1) indications.

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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis. Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product.

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Sanofi wagers $400m on miRecule muscular dystrophy therapy

pharmaphorum

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. The aim will be to identify promising candidates in areas of “unmet patient need.”

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Leqselvi Can Achieve Nearly 90% Scalp Hair Coverage in Severe Alopecia Areata Patients

XTalks

Alopecia areata can be triggered by genetics, immune system dysfunction and environmental factors. It leads to partial or complete hair loss on the scalp and other body parts, with patterns varying from patchy bald spots to total scalp or body hair loss.

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Predicting heart disease from the skin

Scienmag

Jefferson researchers find that the genetic underpinnings of a skin disorder at birth indicate future heart problems PHILADELPHIA – Our skin tells us when we’ve spent too much time in the sun or when the dry air of winter has sucked away too much moisture.

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How to Remove Age Spots: A Comprehensive Guide

Olympian Clinical Research

If you are wondering how to remove age spots, Academic Alliance in Dermatology can help. Genetics, hormonal changes, and certain medications can also play a role in developing age spots. Our team of experienced dermatology providers can help you determine the most suitable treatment plan tailored to your specific needs.

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Digital clinical trials: Trends to watch in 2023

Pharmaceutical Technology

Metabolic disorders are top therapy areas Analysis curated by GlobalData [5] shows the top three therapy areas for DCT adoption in the first quarter of 2022 were metabolic disorders, followed by central nervous system (CNS) and genetic disorders.