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 ...

Antisense Oligonucleotide-based Therapies for Inherited Retinal Diseases: From Mutation to Clinical Trial

March 1st, 2022|Categories: Past Webinars|

Speaker: Alex Garanto, PhD Date: March 10, 2022 Time: 11-12 EST Description: The eye is at the forefront of therapeutic development for its unique properties. Local delivery of ASOs to the retina has shown to be well tolerated and safe. During this webinar, Prof. ...

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 ...

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