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Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053)
SRPT press release – golodirsen NDA acceptance 02 14 19
UT Southwestern Wellstone Symposium
We are excited about the UT Southwestern Wellstone Symposium on March 30! Don’t forget to RSVP by March 1. We’ll see you there! [download id=”13142″ template=”UT Southwestern Wellstone Symposium “]
UT Southwestern President’s Lecture Series – Gene Therapy and Gene Editing
Thursday February 28th at 4pm Dr. Eric Olson is giving a talk at the President’s Lecture at UT Southwestern (South Campus Gooch Auditorium) and will be highlighting the DMD project. Steve […]
Why My Patient Advocacy Organization Is Investing In CRISPR
In last year’s action film “Rampage,” which featured Dwayne “The Rock” Johnson, CRISPR gene-editing technology turns a gorilla, a wolf, and a crocodile into monsters the size of skyscrapers that destroy Chicago. […]
Defying the Odds of Living with Duchenne, Decade After Decade
When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the lucky ones. A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne at […]
NS Pharma Community Letter
We are excited that our industry partner, NS Pharma, has applied for accelertated approval for it’s exon 53 skipping therapy, viltolarsen. We applaud the dedication to the Duchenne community by […]
New Year Letter – Santhera to DMD Community
We’re pleased to share this letter and news from our partner Santhera about their plans for 2019! For more than 10 years, Santhera has been working on treatments for DMD, […]
Two Families Building a Future Through Shared Tragedy
In November 2016, two San Diego families received the same devastating news about their little boys. Four-year-old Yannick Etienne and 5-year-old Marko Filenko were each diagnosed with Duchenne muscular dystrophy, […]
Steroid Use in Duchenne: Expert Dialogue on Latest Data
Healthcare professionals, personal caregivers and family members of those with Duchenne muscular dystrophy gathered to learn about new data and best practices for steroid use in Duchenne at a webinar […]