Beijing MEDPACE Medicine Technology Co.,Ltd.

The primary objective of this study was to evaluate the effectiveness of IP compared with placebo based on the change from baseline in FVC (mL; measured in SSc-ILD study subjects) at the end of the treatment period at Week 52. The secondary objectives of this study were to evaluate the following: ? To evaluate the effectiveness of IP compared with placebo based on the change from baseline at Week 52 in: o Lung involvement as measured by Quantitative ILD (QILD)-Whole Lung (WL) [QILD-WL] on high-resolution computed tomography (HRCT); and o Dyspnea (severity and functional limitation) as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Dyspnea score. ? Overall treatment response as measured by the Composite Response Index for Diffuse SSc-Revised (CRISS) score at Week 52 in diffuse cutaneous SSc study subjects; and ? The safety and tolerability of IP.

The objective of this study was to evaluate the safety, efficacy, and tolerability of long-term dosing of Ataluren 10 mg/kg, 10 mg/kg, and 20 mg/kg in nmDMD subjects who wished to continue treatment after completing a Phase III study (PTC124-GD-041-DMD). The safety profile of ataluren will be described based on the type, frequency, severity, timing, and relationship to ataluren of all adverse events (AEs), laboratory abnormalities, vital signs, electrocardiogram (ECG), echocardiogram, and physical examination findings. The efficacy of ataluren will be evaluated based on change from baseline in upper limb function (PUL), DMD upper limb patient-reported outcome measures (PROMs), spirometry, loss of ambulation ability (LoA), and 6-minute walk test (6MWT).

Part C: Oral Pritelivir (100 mg once daily for up to 28 days) and intermittent intravenous infusion of foscarnet (40 mg/kg every 8 hours or 60 mg/kg every 12 hours, with a minimum of 1 hour injection for up to 28 days) were used to study the efficacy of Pritelivir in immunocompromised subjects with mucocutaneous HSV infection who were ACV-resistant (ACV-R) and resistant/intolerant to foscarnet. Part D: Oral Pritelivir (100 mg once daily for up to 28 days) was used to study the efficacy of Pritelivir in immunocompromised subjects with mucocutaneous HSV infection who were ACV-resistant and resistant/intolerant to foscarnet. Part E: Oral Pritelivir (100 mg once daily for up to 28 days) was used to study the safety of Pritelivir in immunocompromised subjects with mucocutaneous HSV infection who were ACV-sensitive (ACV-S).