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New avenues for rare disease treatment

Drug Target Review

Most of these conditions are genetic in origin and the majority have no effective treatment. One of our drug candidates at Samara activates Transient Receptor Potential Mucolipin 1 (TRPLM1), a protein that’s at the centre of the degradative machinery that’s so key to autophagy.

Disease 113
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Macrocyclization via Ring-Closing Metathesis in Drug Discovery & Process Chemistry

Drug Hunter

Macrocyclic molecules, containing 12 or more atoms in a ring system, are increasingly prevalent in drug discovery, with over 67 macrocyclic drugs approved so far and several notable examples of clinical success highlighted on Drug Hunter recently, including MK-0616 , repotrectinib , and pacritinib.

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How to Get Drugs Into the Brain

Drug Hunter

Obtaining adequate drug exposure in the brain is key to treating CNS diseases effectively. Recently, Dennis Koester gave us a crash course in CNS drug discovery in a Drug Hunter Flash Talk. Why Kp,uu is the Most Important Parameter in CNS Drug Discovery What Influences the Kp,uu of Drugs?

Drugs 206
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Q&A: New approaches are needed to find better cancer drug targets

Broad Institute

At the Broad Institute of MIT and Harvard and elsewhere, researchers are also learning how dependencies affect cancer cells and how they influence each other and contribute to drug resistance. You’d need to disable both genes to see how important they are as a drug target. We miss that combinatorial dependence in our current screens.

Drugs 128
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Building better brain models for Parkinson’s disease and beyond

Drug Target Review

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. This approach has the potential to revolutionise clinical trial design and lead to more effective, personalised treatments.

Disease 52
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CBD Research: A Dive into the Regulations of Cannabis Research

Advarra

However, it’s not legal federally and is considered a Schedule I drug by the U.S. Drug Enforcement Administration (DEA), meaning it has no accepted medical use and a high potential for abuse. The 2018 Farm Bill. The 2018 Farm Bill removed hemp from the Controlled Substance Act. Funding Research on Cannabis.

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Human neuronal cells: possibilities in drug safety testing

Drug Target Review

These cells demonstrate considerable promise for uncovering drug-induced perturbations to neuronal function such as seizure, and their use extends further to sedation, anti-epileptic drug discovery and modelling of neurological diseases.

Drugs 111