Could Artificial Intelligence Provide the Key to Life Saving Medicines?

May 9th, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

Drug discovery is time consuming and expensive, requiring trial and error screening. This means that many people have to wait decades, or a lifetime, to find a treatment for their disease. Others, especially those with rare diseases, may never have hope of a treatment. ...

A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD

April 22nd, 2022|Categories: Perspectives on Current Science|

Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body's muscle tissues to progressively waste away until all mobility is lost. Eventually, the heart muscle becomes fatty and fibrotic, typically leading to heart failure and death by age 30. There is no ...

A New ASO Shows Promise in Treating Dravet Syndrome, a Severe Form of Epilepsy

February 28th, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

 Typically, a single copy of a gene is enough to support normal growth and development but in a small subset of genes, the loss of one copy can cause serious diseases that are often difficult to treat. One of these conditions, Dravet Syndrome, has no ...

A Potent ASO Shows Encouraging Results in A Single-Patient Pilot Study for ALS

February 9th, 2022|Categories: Perspectives on Current Science|

 Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disease in which motor neurons die. Since the motor neurons that die control voluntary muscles, the disease causes great difficulty for those who must live with it. They will experience loss of motor control of the arms ...

Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease

January 1st, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs have proven to be efficient at providing deep reductions in disease causing proteins, and a ...

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