Susan Iannaccone

Myoediting of Duchenne Muscular Dystrophy

National Institute of Child Health and Human Development · P50

PI · $8M · Ut Southwestern Medical Center

IND-Enabling Studies for Aspartylglucosaminuria (AGU) to Support the Initiation of an AAV9/AGA Gene Transfer Clinical Trial

National Institute of Neurological Disorders and Stroke · U01

PI · $631.7K · Ut Southwestern Medical Center

UTSW Clinical Research Site for Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT)

National Institute of Neurological Disorders and Stroke · U24

PI · $1.6M · Ut Southwestern Medical Center

Network of Excellence in Neuroscience Clinical Trial (NEXT)

National Institute of Neurological Disorders and Stroke · U10

PI · $2.2M · Ut Southwestern Medical Center

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

Principal investigator · Recruiting · Phase 1, Phase 2

Elpida Therapeutics SPC

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Principal investigator · Completed · Phase 3

Santhera Pharmaceuticals

Extracellular RNA Biomarkers of Myotonic Dystrophy

Site PI · Recruiting

Massachusetts General Hospital