Drug Discovery World
OCTOBER 5, 2023
A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. Both treatments were able to improve the calcium problems, but the genetic therapy was the most effective.
XTalks
MAY 26, 2023
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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pharmaphorum
OCTOBER 5, 2022
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.”
Drug Discovery World
DECEMBER 19, 2022
This was a life-changing event for Vincenzo,” said Peter Marinkovich, Associate Professor of Dermatology and Director of Stanford Medicine’s Blistering Disease Clinic. One trial participant was Vincenzo Mascoli, aged 22. Now he can bathe and sleep on his back without pain.
Drug Discovery World
DECEMBER 13, 2022
The researchers said the results indicate that rocatinlimab has the potential to change the genetic makeup of a person’s atopic dermatitis for the long term, and possibly help sustain lasting results without continued use of the medication. .
The Pharma Data
MAY 1, 2023
Associate Clinical Professor of Dermatology at the University of California San Francisco (UCSF), Director of Medical Dermatology Consultative Services and Patch Testing for the Palo Alto Foundation Medical Group and lead author on the face and hand analysis.
Scienmag
DECEMBER 10, 2020
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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