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Drug Discovery Research

Snapshot: What are Intrathecal Injections?

Drug delivery into the body can be achieved in several ways, from applying a medicated cream on the skin, to swallowing a pill, to injecting into a muscle or vein. Each route of delivery should at least achieve one thing – getting the drug to the part of the body Read More…

Snapshot: What is the Blood-Brain Barrier?

What is the blood-brain barrier? Blood circulates throughout the body in tubes called blood vessels, delivering oxygen and essential nutrients to different organs. However, not all things that circulate through the body can get into the brain. The blood vessels of the brain are slightly different. Their walls have a Read More…

Targeting protein degradation to alleviate symptoms in MJD

Written by Ambika Tewari   Edited by Brenda Toscano Márquez Trehalose, a natural autophagy inducer shows promise as a therapeutic candidate for MJD/SCA3 Every cell has an elaborate set of surveillance mechanisms to ensure optimal functioning. As proteins are synthesized, errors can occur leading to misfolded proteins. These abnormal proteins can Read More…

A New Use for Old Drugs

Written by Dr. Amy Smith-Dijak Edited by Logan Morrison Basic biology helps identify a new treatment for ataxia Drug design doesn’t always have to start with a blank slate. Sometimes understanding how existing drugs work can help researchers to design new ones, or even to recombine old drugs in new Read More…

Snapshot: What is Omaveloxolone?

March 2023 Update: SKYCLARYS™ (Omaveloxolone) has become the first FDA-approved treatment for Friedreich’s Ataxia. You can learn more about SKYCLARYS™ at this link. This Snapshot was written in May 2020 after initial positive results were shared about Omaveloxolone. A new therapeutic compound shows promise to treat Friedrich’s ataxia. What is Read More…

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