ORCID Profile
0000-0001-8927-2018
Current Organisation
Children's Hospital of Eastern Ontario Research Institute
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Publisher: Elsevier BV
Date: 2018
Publisher: Wiley
Date: 19-01-2011
DOI: 10.1002/MUS.21867
Abstract: Seven children with sciatic neuropathy associated with an underlying neoplasm are reported. Clinical presentation, electrophysiological data, imaging, pathology, and/or autopsy results are described. Pain and weakness, primarily foot drop, were the most common presenting symptoms. The mechanism of sciatic neuropathy was varied and included: nerve infiltration by the adjacent neoplasm (neuroblastoma, rhabdomyosarcoma, and leukemic or lymphomatous infiltration) an expanding, intrinsic neurogenic tumor (perineurioma) or intraoperative stretch injury (osteosarcoma resection). The prognosis for sciatic nerve recovery was good among children who survived their associated cancer. Three children died from the cancer or complications of treatment. One child with perineurioma remained clinically stable, and two children improved after treatment of their neoplasm.
Publisher: Springer Science and Business Media LLC
Date: 21-10-2019
Publisher: Springer Science and Business Media LLC
Date: 03-2021
Publisher: Springer Science and Business Media LLC
Date: 17-06-2022
DOI: 10.1038/S41591-022-01866-4
Abstract: SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report final results for 14 children with two copies of SMN2 , expected to develop spinal muscular atrophy (SMA) type 1. Efficacy was compared with a matched Pediatric Neuromuscular Clinical Research natural-history cohort ( n = 23). All 14 enrolled infants sat independently for ≥30 seconds at any visit ≤18 months (Bayley-III item #26 P 0.001 11 within the normal developmental window). All survived without permanent ventilation at 14 months as per protocol 13 maintained body weight (≥3rd WHO percentile) through 18 months. No child used nutritional or respiratory support. No serious adverse events were considered related to treatment by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for children expected to develop SMA type 1, highlighting the urgency for universal newborn screening.
Publisher: Springer Science and Business Media LLC
Date: 17-06-2022
DOI: 10.1038/S41591-022-01867-3
Abstract: Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated within six postnatal weeks. Of 15 children with three SMN2 copies treated before symptom onset, all stood independently before 24 months ( P 0.0001 14 within normal developmental window), and 14 walked independently ( P 0.0001 11 within normal developmental window). All survived without permanent ventilation at 14 months ten (67%) maintained body weight (≥3rd WHO percentile) without feeding support through 24 months and none required nutritional or respiratory support. No serious adverse events were considered treatment-related by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for presymptomatic infants at risk of SMA type 2, underscoring the urgency of early identification and intervention.
Publisher: Springer Science and Business Media LLC
Date: 31-05-2018
Location: United States of America
Location: Canada
No related grants have been discovered for Hugh McMillan.