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Showing 7 matches for the following: Inventor: "Koeberl, Dwight" ×
Gene therapy for long-term treatment of Pompe disease
Unmet Need Pompe disease is a rare heritable disorder impacting heart and skeletal muscle function with an estimated incidence rate of 1 in 40,000 births in the US. Of these affected births, a third take…
Gene therapy for long-term treatment of GSDIa
Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…
Gene therapy for ATP1A3-Related Neurologic Disorders
Unmet Need Children with Alternating Hemiplegia of Childhood can have episodes of temporary paralysis, dystonia, and severe behavioral problems. The prevalence of AHC is estimated to be 1 in one million to 1 out of…
Clenbuterol Therapy for Amyotrophic Lateral Sclerosis
Unmet Need Amyotrophic lateral sclerosis (ALS) is a debilitating disease with progressive loss of muscle function and inevitable death usually in 3-5 years. There are currently two available therapies on the market which prolong life…
Muscle mass influences upon response to therapy
Duke University is seeking a company interested in commercializing a drug treatment method for enhancing lysosomal storage disease therapy by modulating cell surface receptor expression. Lysosmal storage diseases (LSDs), including Pompe disease, Gaucher disease, Farby…
Method for treating glycogen storage disease
The present disclosure is directed to methods of treating a cytoplasmic glycogen storage disorder, including glycogen storage disease I, glycogen storage disease III, glycogen storage disease IV, and/or conditions associated with a PRKAG2 mutation, by…
Methods of treatment in diseases treated with therapeutic proteins
The present invention relates, in general, to a method of treating patients undergoing enzyme replacement therapy (ERT) or other therapy involving the administration of a proteinaceous therapeutic agent as well gene replacement therapy with non-viral…