Skip to main content

Showing 7 matches for the following: Inventor: "Koeberl, Dwight" ×

Methods of treatment in diseases treated with therapeutic proteins

Methods of treatment in diseases treated with therapeutic proteins

Unmet Need The challenge of antibody formation against protein replacement therapies (PRT) significantly impacts the efficacy of treatments as patients often develop anti-drug antibodies, particularly those who are CRIM-negative, leading to adverse outcomes and limiting…

Read More

Novel Method for Treating Steatosis-Associated Diseases

Novel Method for Treating Steatosis-Associated Diseases

Unmet Need Steatosis-associated diseases are marked by the infiltration of the liver with fatty acids. Liver steatosis occurs in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), as well as rare diseases such as…

Read More

Gene therapy for long-term treatment of Pompe disease

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…

Read More

Gene therapy for long-term treatment of GSDIa

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…

Read More

Gene therapy for ATP1A3-Related Neurologic Disorders

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…

Read More

Clenbuterol Therapy for Amyotrophic Lateral Sclerosis

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…

Read More

Muscle mass influences upon response to therapy

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…

Read More

Have Questions?

Please contact us or subscribe for more opportunities

Stay in Touch with Us