clinical trials

Clinical Trials & Outcome Measures in Duchenne

Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne and Becker. Research is always advancing and evolving, and outcome measures fall into this category, as…

Learn More

September 21, 2023

Entrada Therapeutics starts dosing in ENTR-601-44 Phase 1 trial

Entrada Therapeutics has announced that the first participant has been dosed in its Phase 1 clinical trial evaluating ENTR-601-44, the company’s proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), for the potential treatment of…

Learn More

November 10, 2021

BioMarin shares clinical development update

Late last month BioMarin announced a new pre-clinical candidate for Duchenne, BMN 351. BMN 351 is a third-generation antisense oligonucleotide therapy under investigation for exon 51 skipping that is designed to target a distinct site…

Learn More

November 8, 2021

Recap: Recent Research Updates

Welcome to FALL Time flies! Over the past few months, we have seen a number of announcements regarding drug development, changes to clinical trials, and new trials opening or on the horizon. For many, it…

Learn More

Take Part in the Duchenne Steroid Patient Experience Survey

Patient experience data is a critical tool for advancing drug development in Duchenne and for regulatory decision making. Steroids have been part of care standards for over two decades, however, there is a lack of…

Learn More

November 21, 2020

NEW – Fall 2020 Survey: Impact of the COVID-19 Pandemic on Duchenne

PPMD has launched our second COVID-19 Pandemic Impact Survey related to your experiences during the pandemic for accessing care, trials, and approved therapies. Thank you to everyone who completed our first survey earlier in the spring….

Learn More

Have You Participated in a Clinical Trial That Was Terminated?

In an effort to learn more about the patient and family experience around clinical trials that have stopped or were terminated, PPMD and Duchenne UK have collaborated on a survey to learn more about your…

Learn More

Survey: Impact of the COVID-19 Pandemic on Duchenne

Today, PPMD launched a survey to gain insight directly from the Duchenne and Becker community on the impact of the COVID-19 pandemic related to care, clinical trials, and access to FDA approved therapies. This information…

Learn More

Join Our Mailing List