News Features

Patient Voices

Sarepta_Vassigh_Twins

Video: Scholarship Recipients, and Twins, Austin and Connor Travel Route 79 Together

Advocates & Innovators

Roots of innovation

Roots of innovation: From science fairs to the leading edge of precision genetic medicine

Advocates & Innovators

Crane

Navy veteran, lifelong learner, dedicated Duchenne Nurse Educator: Meet John Crane

Patient Voices

Landry

Wearable devices and the future of rare disease clinical trials: Landry’s story

Advocates & Innovators

sky newsroom

Awards & Recognitions: Sarepta recognized for innovation and industry leadership

Inside the Science

Journey Trial Image

Journey to better understanding of LGMD: Q&A on the early findings of a new natural history study

Culture & Community

Shawn Newsroom

Continuing a family legacy: Meet Sarepta colleague and Duchenne advocate Shawn Marlow

Patient Voices

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When time is muscle: One dad shares his story to advocate for Duchenne newborn screening

Advocates & Innovators

guardians of the last mile

Guardians of the last mile: Ensuring smooth delivery and a seamless patient experience

Advocates & Innovators

Kate & Katherine

A tale of two interns: Meet Kate & Katherine, rare disease advocates and members of Sarepta’s Patient Affairs team

Inside the Science

GT dosing newsroom teaser

What’s in a dose? Gene therapy dosing explained (Video)

Advocates & Innovators

Stefanie Mason

Seeing hope at genetic medicine's frontier: Meet Stefanie Mason, M.D.

Inside the Science

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Why are steroids used in gene therapy for neuromuscular disease?

Advocates & Innovators

Danielle

A passion for patients, an influential mentor and a change in plans: Meet Danielle Griffin

Patient Voices

Picture of Rachel Alvarez

Creating pathways to LGMD genetic diagnosis: Spotlight on LGMD Grant Award recipient

Advocates & Innovators

picture of Eddie

Ensuring the safety of clinical trial participants: Meet Eddie Darton, M.D.

Advocates & Innovators

Rachel Potter

Dragging tomorrow into today: Meet Rachael Potter, Ph.D.

Inside the Science

external controls

Understanding external controls and their importance to clinical trials for rare diseases

Inside the Science

meaningful measurements

Meaningful measure: Understanding the NSAA, an important Duchenne functional-assessment tool

Culture & Community

2022 Interns

What’d you do this summer? Our summer interns share lessons learned

Culture & Community

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Pride proud: What belonging feels like and why representation matters

Advocates & Innovators

photo of Ian M. Estepan

A conversation with CFO Ian Estepan, recipient of the 2022 CFO of the Year award from the Boston Business Journal

Inside the Science

antibodies

Seroprevalence: What patients should know about pre-existing antibodies to gene therapy

Inside the Science

Registries image

Patient registries: A catalyst for developing new therapies for rare diseases

Inside the Science

Endpoints image

Measurements used in clinical trials for Duchenne muscular dystrophy – an overview

Advocates & Innovators

ami kabadi

How a collaboration in the lab led to a seat at the table in Sarepta’s Gene Editing Innovation Center

Inside the Science

GT-FAQ

Welcome to GT‐FAQ!

Advocates & Innovators

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Expanding access to newborn screening for those with rare diseases

Patient Voices

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Patiently waiting

Advocates & Innovators

Jungels

From art to science: How one Sarepta scientist brings arts into the lab