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1 Disease |  |
0 Trials |
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0 Trials |  |
8 News |  |
- | AAV1-Follistatin / Milo Biotechnology, Tamoxis (tamoxifen) / Bioprofarma
Preclinical, Journal: AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD. (Pubmed Central) - Nov 14, 2019
AAV1.Follistatin significantly increased TIC-DUX4 muscle mass and strength even in the presence of DUX4 expression, suggesting that myostatin inhibition may be a promising approach to treat FSHD-associated weakness. We conclude that TIC-DUX4 mice are a relevant model to study DUX4 toxicity and, importantly, are useful in therapeutic development studies for FSHD.
- | AAV1-Follistatin / Milo Biotechnology
Trial completion, Trial primary completion date: Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis (clinicaltrials.gov) - Nov 24, 2017
P1, N=15, Completed, Sponsor: Nationwide Children's Hospital
We conclude that TIC-DUX4 mice are a relevant model to study DUX4 toxicity and, importantly, are useful in therapeutic development studies for FSHD. Active, not recruiting --> Completed | Trial primary completion date: Jul 2017 --> Oct 2017
- | AAV1-Follistatin / Milo Biotechnology
Trial completion, Trial primary completion date: Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - Nov 24, 2017
P1/2, N=6, Completed, Sponsor: Jerry R. Mendell
Active, not recruiting --> Completed | Trial primary completion date: Jul 2017 --> Oct 2017 Active, not recruiting --> Completed | Trial primary completion date: Jul 2017 --> Nov 2017
- | AAV1-Follistatin / Milo Biotechnology
Enrollment closed, Trial primary completion date: Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - Mar 29, 2017
P1/2, N=6, Active, not recruiting, Sponsor: Jerry R. Mendell
Active, not recruiting --> Completed | Trial primary completion date: Jul 2017 --> Nov 2017 Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Jan 2017 --> Jul 2017
- AAV1-Follistatin / Milo Biotechnology
Enrollment closed, Trial primary completion date: Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis (clinicaltrials.gov) - Jan 27, 2016
P1, N=15, Active, not recruiting, Sponsor: Nationwide Children's Hospital
Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Jan 2017 --> Jul 2017 Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Dec 2015 --> Jul 2017
- AAV1-Follistatin / Milo Biotechnology
New P1/2 trial: Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - Feb 3, 2015
P1/2, N=6, Enrolling by invitation, Sponsor: Jerry R. Mendell
- AAV1-Follistatin / Milo Biotechnology
New P1 trial: Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis (clinicaltrials.gov) - Jan 24, 2012
P1, N=15, Enrolling by invitation, Sponsor: Nationwide Children's Hospital