S. Vacca
Addgene(US)
Publications by Year
Research Areas
Muscle Physiology and Disorders, Genetic Neurodegenerative Diseases, Delphi Technique in Research, Health Systems, Economic Evaluations, Quality of Life, Cardiomyopathy and Myosin Studies
Most-Cited Works
- → PGN-EDO51, an Enhanced Delivery Oligonucleotide (EDO) for the Treatment of Duchenne Muscular Dystrophy (DMD): Results of a Phase 1 Study in Healthy Volunteers (P3-8.004)(2023)6 cited
- → 461P Evaluation of PGN-EDODM1: FREEDOM-DM1 and FREEDOM2-DM1 clinical trials in myotonic dystrophy type 1(2024)2 cited
- → P44 Phase 1 study of PGN-EDO51 demonstrates tolerability, delivery and high levels of exon skipping for treatment of Duchenne muscular dystrophy (DMD)(2023)2 cited
- → 403P CONNECT1-EDO51: a 12-week open-label Phase 2 study to evaluate PGN-EDO51 safety and efficacy in people with Duchenne amenable to exon 51 skipping(2024)1 cited
- → Positive results from a first-in-human study supporting continued development of PGN-EDO51 for the treatment of duchenne muscular dystrophy (DMD)(2023)1 cited
- → 404P CONNECT2-EDO51: a Phase 2 placebo-controlled study to evaluate PGN-EDO51 safety and efficacy in people with Duchenne amenable to exon 51 skipping(2024)
- → P26 CONNECT-EDO51: Trial designs to support the development of PGN-EDO51 for Duchenne Muscular dystrophy amenable to exon 51 skipping(2023)
- → Design of a phase 2 clinical program for the development of PGN-EDO51 in participants with duchenne muscular dystrophy (DMD)(2023)
- → Design of a Phase 1, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (DM1) (P6-8.004)(2023)
- → P391 Phase 1 study to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of PGN-EDODM1 in adults with myotonic dystrophy type 1 (DM1)(2023)