AMT-110 / uniQure - LARVOL DELTA

Reliability of corticospinal excitability and intracortical inhibition in biceps femoris during different contraction modes. (PubMed, Eur J Neurosci) - "Single-pulse outcomes measured during isometric contractions (AURC, AMT 110%, AMT 120%, AMT 130%, AMT 150%, AMT 170%) demonstrated fair to excellent reliability (ICC range, 0.51 to 0.92; CV%, 21 to 37%), whereas SICI, LICI and ICF demonstrated good to excellent reliability (ICC range, 0.62 to 0.80; CV%, 19 to 42%). This study found that both single- and paired-pulse TMS outcomes can be measured from the biceps femoris muscle across all contraction modes with fair to excellent reliability. However, coefficient of variation values were typically greater than the smallest worthwhile change which may make tracking physiological changes in these variables difficult without moderate to large effect sizes."

Journal • Immunology

Intracerebral gene therapy in 4 children with Sanfilippo B syndrome : 5.5 years follow-up results. (PubMed, Hum Gene Ther) - "Treatment consisted in 16 intra-cerebral and cerebellar deposits of a recombinant adeno-associated viral vector encoding human -N-acetylglucosaminidase (rAAV2/5-hNAGLU) plus immunosuppression...Cognitive benefit was not confirmed in the 3 oldest patients. Milder disease in the youngest patient support further investigations of adeno-associated-vector-mediated intracerebral gene therapy in Sanfilippo B."

Clinical • Journal • Gene Therapies • Immunology • Lysosomal Storage Diseases

QUILT-3.039: Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome (clinicaltrials.gov) - P1/2; N=4; Completed; Sponsor: UniQure Biopharma B.V.; Active, not recruiting ➔ Completed

Clinical • Trial completion