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An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…

A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan-N-sulfatase (rhHNS) Intrathecal Administration via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…

An open-label phase 2A study to investigate drug-drug interactions between AT1001 (migalastat hydrochloride) and agalsidase in subjects with Fabry disease.

The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…

An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease

Long-term safety and pharmacokinetics (PK) of neoGAA

Short-term safety and efficacy of ketohexokinase inhibition in patients with hereditary fructose intolerance

To study the effects of PF-06835919 on fructose tolerance and intrahepatic lipid content in patients with HFI.

An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia

PrimaryTo characterize the effect of 90 and 180 days of subcutaneous ALN-PCSSC on the percentage change from Day 1 inlow-density lipoprotein cholesterol (LDL-C) in subjects with homozygous familial hypercholesterolemiaSecondary* To assess the effect…