BioMarin Introduces kNOWyourDuchenne Program to Guide DMD Patients Into Ongoing Clinical Trials

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

BioMarin Pharmaceutical, Inc., a company that develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions, has officially launched kNOWyourDuchenne, a program dedicated to assisting families and clinicians gain access to genetic testing for patients with Duchenne muscular dystrophy (DMD).

This first-of-its-kind program will work to pinpoint specific genetic mutations linked to DMD, which can serve as a guide for healthcare providers to recommend promising treatments currently under development. Today’s genetic tests lack the extra step of having enough information on where DMD patients can possibly turn to and avail of promising, investigational treatments that are a fit with their specific genes.

“Creating the kNOWyourDuchenne program fits with BioMarin’s mission to provide highly personalized support and services to patients,” said Chuck Bucklar, Vice President Commercial Operations. “Every family with Duchenne has specific needs unique to them, and BioMarin has a decade of experience in tailoring support services based on individual need. The first step to provide support to patients with Duchenne is to make sure that each person with Duchenne is tested and has access to an independent genetic counselor to provide an accurate interpretation of the test results.”

Through the kNOWyourDuchenne program, the company will be providing genetic testing and interpretation at no cost to eligible DMD patients. “It is important for children and their families with Duchenne muscular dystrophy to know their mutation in order to understand if they qualify for a clinical trial, or to know if they might be candidates for future treatments. Having access to an expert to interpret the genetic test results is critical to determine the most appropriate care,” said Patrick Pallansch, Vice President, Medical Affairs.

Today, DMD remains incurable, but a good number of promising therapies are still in clinical stage development – all in need of willing and qualified participants. Genetic tests similar to what BioMarin offers should place vital information and guidance in the hands of DMD patients, their families, and attending physicians for the sake of giving patients a shot at starting cutting-edge treatment and at the same time, help pharmaceutical companies and researchers attain faster marketing approval for treatments.

BioMarin has an established track record of providing support services to patients, including diagnostics for diseases in which the company is developing treatments. The kNOWyourDuchenne program builds on BioMarin’s expertise in personalized patient support services.

To know more about kNOWyourDuchenne and to request access to genetic interpretation and testing services, go to or call 1-844-DUCHENNE (382-4366).