pharmaphorum

OCTOBER 5, 2022

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 101

RNA Genetics Dermatology Antibody 101

Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

Drug Discovery World

FEBRUARY 28, 2023

mRNA enables the delivery of a transiently expressed genetic molecule that is translated into a target protein using the machinery of the host cell. Finally, dosing and timing may be controlled simply by adjusting the amount of mRNA delivered to the cells, rather than relying on inducible systems that are integrated into the genome.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

OCTOBER 28, 2020

All patients experienced numerical increases in the sub-scale growth scores of expressive and/or receptive communication of the Bayley Scales of Infant and Toddler Development (Bayley-4) domains, and three patients showed improvements in the Observed Reported Communication Ability (ORCA) measure of expressive, receptive, and pragmatic communication.

Protein 40

Protein Production Development Drugs 40