Sarepta Therapeutics’ Post

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Today we announced an important development regarding FDA approval of our gene therapy. Watch to hear a message from our CEO, Doug Ingram: 

Dimitri Kaye

Senior Talent Acquisition Specialist for Diagnostic & Genomics Segment at Bio-Techne.

1mo

Exciting to see how far Sarepta has come from a mom and pop shop, based out of Washington State, to being the leader in DMD care. #Thankful

Jeff Lansman

Professor Emeritus of Cellular & Molecular Pharmacology, UCSF School of Medicine

1mo

This drug was rejected by every FDA scientific advisory review: it was found not to be curative nor alter the course of this tragic disease; it met no clinical endpoints; it led to only small increases in surrogate endpoint, which is expression of dystrophin minigene protein that bears minor resemblance to the Becker’s gene product; serious adverse effects including reactions to adenovirus vector, which precludes therapy from future, efficacious gene therapy; and serious liver damage and other concerns. Meanwhile, it costs millions of dollars for treatment. (from report): “…Three FDA review teams and two top officials recommended Sarepta’s application be rejected due to insufficient and conflicting clinical data. They were overruled by Marks, head of the FDA center that reviews gene therapies, who found the results supportive enough to broaden Elevidys’ label. It’s now cleared for Duchenne patients over the age of 4 with mutations to a specific gene, regardless of whether they can still walk.”

Daniel Alaniz

Implementation Manager at Cryoport

1mo

Fantastic news! It is wonderful to see that the range of patients who can benefit has expanded, bringing more hope to even more families! ❤️

Jennifer Fairbanks Braverman 🎗️

Improving the Quality of Life Through Impactful Leadership | Life Science | jbraverman@wittkieffer.com

1mo

Wonderful news for patients and a hearty congratulations to the dedication of so many that made this possible! Keep shining on! ❤️

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Sarah Kasner, MBA, CCP

Principal at SullivanCotter

1mo

“Today is a good day” Precisely. Congrats again, such incredible progress.

Michael Middendorp

Associate Financial Advisor at Edward Jones | MBA Alumnus at CSU Chico | DMD Advocate

1mo

Today is definitely a good day! Thank you and congrats to all who were involved with this!

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Kristen Pearse

Director - Global Alliance Management at FujiFilm Diosynth Biotechnologies

1mo

I am overjoyed to hear this news! So many lives will be changed. Congrats!

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Tara Ziegler

Biopharma Business Development | Helping drug developers through quality assays

1mo

This is wonderful news! Congratulations to affected families and the entire Sarepta team.

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Congratulations!!! A great day for patients!!!

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