New gene therapy shows promise for kids with rare muscle disease
NCT ID NCT05089656
First seen Jan 09, 2026 · Last updated May 16, 2026 · Updated 16 times
Summary
This study tested a one-time gene therapy called OAV101 in 126 children aged 2 to 18 with type 2 spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. Participants received either the therapy or a sham procedure. The goal was to see if the treatment improves motor function, like sitting and moving arms, compared to no treatment. The study is complete, and results will help determine if this therapy can slow or improve the disease.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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AIIMS, Ansari Nagar
New Delhi, New Delhi, 110029, India
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507, United States
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Children's Specialty Group/CHKD
Norfolk, Virginia, 23507, United States
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Clinic for Special Children
Strasburg, Pennsylvania, 17579, United States
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Connecticut Children's Medical Center
Farmington, Connecticut, 06032, United States
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Hospital Civil De Guadalajara Fray Antonio Alcalde
Guadalajara, Jalisco, 44280, Mexico
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Kaohsiung Medical University Hospital
Kaohsiung City, 80756, Taiwan
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National Children's Hospital
Hanoi, 100000, Vietnam
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Novartis Investigative Site
Curitiba, Paraná, 81520-060, Brazil
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Novartis Investigative Site
São Paulo, São Paulo, 05403 000, Brazil
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Novartis Investigative Site
Beijing, Beijing Municipality, 100034, China
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Novartis Investigative Site
Chongqing, Chongqing Municipality, 400010, China
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Novartis Investigative Site
Guangzhou, Guangdong, 510623, China
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Novartis Investigative Site
Shenzhen, Guangdong, 518034, China
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Novartis Investigative Site
Chengdu, Sichuan, 610041, China
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Novartis Investigative Site
Hangzhou, Zhejiang, 310052, China
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Novartis Investigative Site
Beijing, 100069, China
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Novartis Investigative Site
Beijing, 100730, China
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Novartis Investigative Site
Copenhagen, 2100 O, Denmark
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Novartis Investigative Site
New Delhi, National Capital Territory of Delhi, 110029, India
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Novartis Investigative Site
New Delhi, National Capital Territory of Delhi, 110060, India
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Novartis Investigative Site
Kolkata, West Bengal, 700094, India
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Novartis Investigative Site
Hyderabad, 500034, India
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Novartis Investigative Site
Mumbai, 400016, India
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Novartis Investigative Site
Kuala Lumpur, 50300, Malaysia
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Novartis Investigative Site
Kuala Lumpur, 59100, Malaysia
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Novartis Investigative Site
Guadalajara, Jalisco, 44280, Mexico
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Novartis Investigative Site
Mexico City, Mexico City, 06720, Mexico
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Novartis Investigative Site
Riyadh, 11211, Saudi Arabia
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Novartis Investigative Site
Singapore, 119074, Singapore
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Novartis Investigative Site
Cape Town, 7925, South Africa
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Novartis Investigative Site
Kaohsiung City, 80756, Taiwan
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Novartis Investigative Site
Bangkok, 10700, Thailand
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Novartis Investigative Site
Hanoi, 100000, Vietnam
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P.D. Hinduja National Hospital & MRC
Mumbai, 400016, India
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Paediatric Neurology
Copenhagen, 2100 O, Denmark
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Peking University First Hospital
Beijing, 100034, China
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Red Cross War Memorial Childrens Hospital
Cape Town, 7925, South Africa
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Sir Ganga Ram Hospital
New Delhi, National Capital Territory of Delhi, 110 060, India
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Siriraj Hospital
Bangkok, 10700, Thailand
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St Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Conditions
Explore the condition pages connected to this study.