4 results
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…
All efficacy study objectives will be assessed in the pediatric AS study population of subjects who are 4 to 12 years old. All safety study objectives will be assessed in the pediatric AS study population of subjects who are 2 to 12 years old.…
The primary objective of this study is to evaluate the long-term safety and tolerability of OV101 in individuals with AS assessed by the incidence and severity of AEs and SAEs in subjects who are at least 2 years old.The secondary objectives of this…
Primary: • Assessment of the safety and tolerability of PLN-74809 Secondary:• Assessment of pharmacokinetics (PK) of PLN-74809 Exploratory:• Assessment of change from baseline in forced vital capacity (FVC) • Assessment of change from baseline in…