More than 40 rare neuromuscular trials, in indications including, but not limited to, DMD (ambulatory and non-ambulatory), SMA, ALS, Myasthenia Gravis, CIDP and FSHD
Genetic medicine trials (in vivo & ex vitro), including AAV, lentiviral vectors and gene editing studies, with delivery via various routes of administration, including directly into the CNS
Building highly patient-centric protocols, that incorporate patient voice and decentralized trial technologies
