Genetics Nurses Protein 
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.
XTalks
MAY 12, 2023
Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.
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The Pharma Data
APRIL 8, 2021
Roche will present data on the increased use of home nursing capabilities in the Phase III GRADUATE studies of gantenerumab during the COVID-19 pandemic, which enabled home-bound trial participants to continue dosing to maintain medicine exposure. Alzheimer’s Disease (AD). We expect data from the studies in 2022. Huntington’s Disease (HD).
The Pharma Data
DECEMBER 17, 2020
To develop this novel medicine, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.
XTalks
AUGUST 30, 2022
What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? The return of cell therapy-iPSCs (induced pluripotent stem cells) and genetically altered delivery cells. According to Dr. Vornov, there is plenty of evidence that at least some therapeutic antibodies can reach extracellular CNS targets.
The Pharma Data
DECEMBER 3, 2020
Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics. Genetics / 12A. –( BUSINESS WIRE )– Stoke Therapeutics , Inc.
pharmaphorum
DECEMBER 23, 2022
This alone causes expense for cash-strapped hospitals and puts pressure on nursing and medical staff while they manage the side effects. Inobrodib works by targeting twin cancer proteins p300 and CBP, which impacts the expression of cancer drivers including MYC and IRF4. Side effects and cost implications.
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