Publications by authors named "Y S Wasfi"
Article Synopsis
- The PROPEL study compared a new therapy, cipaglucosidase alfa + miglustat (cipa + mig), to the standard treatment, alglucosidase alfa + placebo (alg + pbo), for adults with late-onset Pompe disease (LOPD).
- An ongoing open-label extension study is assessing the long-term safety and effectiveness of cipa + mig, focusing on various outcome measures like walking distance and lung function.
- After 104 weeks, patients on cipa + mig showed maintained improvements in walking distance and biomarkers with minimal safety concerns, indicating potential long-term benefits for those with LOPD.
Article Synopsis
- Cipaglucosidase alfa plus miglustat (cipa + mig) is a new two-part treatment for Pompe disease, assessed in the Phase I/II ATB200-02 study over 48 months.
- The study involved four adult groups, including both ambulating and non-ambulating patients, who received specific doses of cipa and mig biweekly.
- Results showed improvements in walking distances and respiratory capacity, especially in ERT-naïve patients, with the treatment generally well tolerated and a safety profile similar to existing therapies.
Article Synopsis
- This study investigates the genetic factors contributing to sarcoidosis susceptibility by examining novel alleles and the role of HLA (human leukocyte antigen) alleles in both European and African American populations.* -
- A genome-wide analysis of 1335 sarcoidosis cases and 1264 controls from European descent, along with findings from an African American cohort, identified 49 significant SNPs (single nucleotide polymorphisms) linked to the disease.* -
- The research highlights the importance of specific HLA alleles in the disease's development, noting significant associations between these genetic elements and sarcoidosis, reinforcing the idea that HLA class II genes play a critical role in the disease's pathogenesis.*
Background: Long-term evaluation is required to confirm the safety profile of newer biologic agents.
Objectives: To report on pooled safety data from the ongoing VOYAGE 1 (NCT02207231) and VOYAGE 2 (NCT02207244) trials through 100 weeks of follow-up.
Methods: Patients were randomized to either guselkumab 100 mg at weeks 0 and 4 and every 8 weeks thereafter; placebo at weeks 0, 4, 12 followed by guselkumab 100 mg at weeks 16 and 20 and every 8 weeks thereafter; or adalimumab 80 mg at week 0, 40 mg at week 1, and 40 mg every 2 weeks thereafter.
Importance: Psoriasis of the scalp, palms and/or soles, and nails is challenging to treat.
Objective: To evaluate the effect of guselkumab on psoriasis in specific body regions.
Design, Setting, And Participants: VOYAGE 1 and VOYAGE 2 were, double-blind, placebo- and adalimumab-controlled studies of guselkumab conducted at 101 and 115 global sites, respectively, from November 3, 2014, to May 19, 2016.