Exon Skipping Therapies

About us

We do not want children to die from a disease that can be cured. We want to give hope to families and ensure that all children with DMD are treated and regain their health.

Latest Articles

Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease

Research February 4, 2025 0

Cumberland Pharmaceuticals, a specialty pharmaceutical company with development efforts...

Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201

Research February 3, 2025 0

ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical...

Brazilian Children Over 7 and Their Families Demand Elevidys Gene Therapy from Anvisa

DMD February 2, 2025 2

ELEVIDYS, the only currently approved gene therapy for DMD,...

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

DMD December 26, 2024 1

A genetic condition known as Duchenne muscular dystrophy (DMD)...

Cures of Duchenne (List of All Researches)

Research September 20, 2024 7

Dystrophin Gene Therapy MechanismCompanyDrugStatus Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene...

Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy

Research October 10, 2024 0

Capricor Therapeutics, a biotechnology company developing transformative cell and...

Exon Skipping Therapies

Categories

Must Read

Nutrition Guideline for Duchenne Muscular Dystrophy: Foods to Eat and Avoid

Plans for DYNE-101 in DM1 and DYNE-251 in DMD Are Announced by Dyne Therapeutics

Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne