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Acute Nutritional Ketosis in VLCAD Deficiency: testing the metabolic basis for therapeutic use

To test if acute NK boosts muscle mitochondrial function in vivo in patients with VLCADD in order to establish a rational basis for therapeutic use in this disorder. A secondary objective is to gather data to test the working hypothesis that the…

A Study to Evaluate the Efficacy and Safety of GFT505 once daily on Steatohepatitis in Patients with Non-Alcoholic Steatohepatitis (NASH).
A Multicentre, Randomized, Double Blind, Placebo-Controlled study, with an adaptive design to allow for initial GFT505 80mg dosing versus placebo, followed by a second phase including GFT505 120mg dose, after review of 6-month safety analysis of the 80mg data on at least 50% of patients.

The primary objectives of this study are: - To evaluate the efficacy of GFT505 80mg and GFT505 120mg once daily (according to adaptive design, after 6-month interim safety analysis of the 80mg data on at least 50% of patients) for 52 weeks versus…

A Randomized, Controlled, Open-label, Multicenter, Phase IIb
Safety and Efficacy Study of HGT-1410 (Recombinant Human
Heparan N Sulfatase) Administration via an Intrathecal Drug
Delivery Device in Pediatric Patients with Early Stage
Mucopolysaccharidosis Type IIIA Disease

The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…