Science-Based Medicine

Hope, Evidence, and Hard Questions in Duchenne Muscular Dystrophy

I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical ...
Nature

Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP–AON complexes

Duchenne muscular dystrophy (DMD) is an inherited X-linked degenerative muscular disorder predominantly caused by frame-disrupting mutations arising from gross rearrangements in the dystrophin gene. 1 ...
Nature

Restoration of all dystrophin protein interactions by functional domains in trans does not rescue dystrophy

Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for ...
Yahoo Finance

Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin and Clinical Outcomes

CAMBRIDGE, MA--(Marketwired - Apr 15, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding a ...
EurekAlert!

Discovery of new form of dystrophin protein could lead to therapy for some DMD patients

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. Studies of the new protein ...

Connor's Story: Shaker Heights teen may hold the key to life-changing Muscular Dystrophy research, but threatened cuts put its future at risk

A 3News and Signal Cleveland report reveals how proposed NIH cuts could threaten critical research, as well as the young people who depend on it.
MedPage Today

Eteplirsen Boosts Dystrophin, Function in DMD

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window BOSTON -- A ...
Forbes

A Gene Therapy Appears To Replace Missing Protein In Muscular Dystrophy Patients

An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, ...
Yahoo Finance

Global Dystrophin Market to Reach USD 30.22 Billion by 2028 - Heavy Research & Development to Boost the Market Demand – Vantage Market Research

WASHINGTON, Feb. 16, 2022 (GLOBE NEWSWIRE) -- The Global Dystrophin Market size is expected to reach USD 30.22 Billion by 2028, exhibiting a Compound Annual Growth Rate (CAGR) of 45.9% during the ...
Labroots

Webinar: Dystrophin Quantification in Preclinical and Clinical Settings

Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. Muscle fibers lacking dystrophin are more sensitive to damage and over time ...
Medindia

Immunity to Dystrophin Revealed in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy has revealed a challenge to researchers as an immune reaction to the defective muscle protein, dystrophin, makes it difficult to strenghten patients' muscles. Duchenne ...