Genetics in Medicine Open (Jan 2023)
P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study*
- Erin O’Rourke,
- Jerry R. Mendell,
- Zarife Sahenk,
- Kelly J. Lehman,
- Linda P. Lowes,
- Natalie F. Reash,
- Megan Iammarino,
- Lindsay Alfano,
- Sarah Lewis,
- Kathleen Church,
- Richard Shell,
- Rachael A. Potter,
- Danielle Griffin,
- Mark J. Hogan,
- Shufang Wang,
- Stefanie Mason,
- Eddie Darton,
- Louise Rodino-Klapac
Affiliations
- Erin O’Rourke
- Sarepta Therapeutics, Inc.
- Jerry R. Mendell
- Center for Gene Therapy, Nationwide Children’s Hospital
- Zarife Sahenk
- Center for Gene Therapy, Nationwide Children’s Hospital
- Kelly J. Lehman
- Center for Gene Therapy, Nationwide Children’s Hospital
- Linda P. Lowes
- Center for Gene Therapy, Nationwide Children’s Hospital
- Natalie F. Reash
- Center for Gene Therapy, Nationwide Children’s Hospital
- Megan Iammarino
- Center for Gene Therapy, Nationwide Children’s Hospital
- Lindsay Alfano
- Center for Gene Therapy, Nationwide Children’s Hospital
- Sarah Lewis
- Sarepta Therapeutics, Inc.
- Kathleen Church
- Center for Gene Therapy, Nationwide Children’s Hospital
- Richard Shell
- Center for Gene Therapy, Nationwide Children’s Hospital
- Rachael A. Potter
- Sarepta Therapeutics, Inc.
- Danielle Griffin
- Sarepta Therapeutics, Inc.
- Mark J. Hogan
- Center for Gene Therapy, Nationwide Children’s Hospital
- Shufang Wang
- Sarepta Therapeutics, Inc.
- Stefanie Mason
- Sarepta Therapeutics, Inc.
- Eddie Darton
- Sarepta Therapeutics, Inc.
- Louise Rodino-Klapac
- Sarepta Therapeutics, Inc.
- Journal volume & issue
-
Vol. 1,
no. 1
p. 100342
