Equitable Access to Disease-Modifying Therapies for Canadian Children with SMA and Four SMN2 Copies

被引：1

作者：

McMillan, Hugh J. ^{[1
]}

Gonorazky, Hernan ^{[2
]}

Campbell, Craig ^{[3
]}

Chrestian, Nicolas ^{[4
]}

Crone, Megan ^{[5
]}

Dowling, James J. ^{[2
]}

Joyal, Kristina ^{[6
,7
]}

Kolski, Hanna ^{[8
,9
]}

Leung, Ed ^{[6
]}

Mackenzie, Alex ^{[1
]}

Mah, Jean K. ^{[5
]}

McAdam, Laura ^{[10
]}

Nigro, Elisa ^{[2
]}

Nguyen, Cam-Tu ^{[13
]}

Oskoui, Maryam ^{[11
,12
,14
]}

Poulin, Chantal ^{[11
,12
]}

Sheriko, Jordan ^{[15
]}

Tarnopolsky, Mark ^{[16
]}

Vajsar, Jiri ^{[2
]}

Yaworski, Amanda ^{[1
]}

Selby, Kathryn ^{[17
]}

机构：

[1] Univ Ottawa, Childrens Hosp Eastern Ontario, Res Inst, Dept Pediat, Ottawa, ON, Canada

[2] Univ Toronto, Hosp Sick Children, Dept Pediat, Toronto, ON, Canada

[3] Univ Western, Childrens Hosp Western Ontario, Schulich Sch Med, Dept Pediat Epidemiol & Clin Neurol Sci, London, ON, Canada

[4] Univ Laval, Ctr Hosp Mere Enfant Soleil, Dept Pediat, Quebec City, PQ, Canada

[5] Univ Calgary, Alberta Childrens Hosp, Res Inst, Cumming Sch Med, Calgary, AB, Canada

[6] Hlth Sci Ctr, Dept Pediat, Winnipeg, MB, Canada

[7] Jim Pattison Childrens Hosp, Dept Pediat, Saskatoon, SK, Canada

[8] Univ Alberta, Stollery Childrens Hosp, Dept Pediat, Edmonton, AB, Canada

[9] Univ Alberta, Glenrose Rehabil Hosp, Edmonton, AB, Canada

[10] Univ Toronto, Holland Bloorview Kids Rehabil Hosp, Dept Pediat, Div Dev Pediat, Toronto, ON, Canada

[11] McGill Univ, Dept Pediat, Montreal, PQ, Canada

[12] McGill Univ, Dept Neurol & Neurosurg, Montreal, PQ, Canada

[13] Univ Montreal, Ctr Hosp Univ St Justine, Dept Pediat, Montreal, PQ, Canada

[14] McGill Univ Hlth Ctr, Res Inst, Ctr Outcomes Res & Evaluat, Montreal, PQ, Canada

[15] Dalhousie Univ, Dept Pediat & Med, IWK Hlth, Halifax, NS, Canada

[16] McMaster Univ, McMaster Childrens Hosp, Med Ctr, Hamilton, ON, Canada

[17] Univ British Columbia, BC Childrens Hosp, Dept Pediat, Div Neurol, Vancouver, BC, Canada

来源：

CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES | 2025年 / 52卷 / 03期

关键词：

spinal muscular atrophy; neonatal screening; survival motor neuron protein;

D O I：

10.1017/cjn.2024.319

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

引用

页码：543 / 545

页数：3

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