The PPMD Mission

 

Parent Project Muscular Dystrophy’s mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community.

 

Objectives

 

Research

• To identify and aggressively fund the most promising near and long-term Duchenne research and cutting-edge therapies.

• To stimulate new research to ensure that the therapeutic pipeline is rich with opportunity.

• To encourage and support the pharmaceutical industry to maximize their investment in Duchenne.

 

Advocacy

•To ensure the patient’s voice is heard in Washington, DC, so policies lead to improvements in the lives of families affected by Duchenne and reflect the needs of the whole community.

• To work with the National Institutes of Health and other agencies to ensure that Duchenne research and clinical trials remain a high priority.

• To work with federal regulatory agencies so they address Duchenne-specific concerns in their decision-making.

 

Care

• To identify gaps in care for young men with Duchenne and work toward solutions.

• To work with clinicians and other health care professionals across the globe to ensure all Duchenne patients have access to optimal care.

 

Community

• To provide a supportive environment in which people affected by Duchenne can share needs, concerns, and common experiences.

• To work collaboratively with stakeholders who make up the Duchenne population and to participate actively and effectively in the international Duchenne community.

 

Education

• To increase recognition of muscle weakness among healthcare professionals and promote early diagnosis.

• To share up-to-date information about treatment and care options with all members of the Duchenne community.

• To raise awareness about Duchenne and provide educational materials to the global community.

Values

 

Our work and relationships are driven by several deeply held principles:

 

We value…

 

Family

Young men with Duchenne and their families are at the heart of everything we do, and they always come first.

 

Impact

We believe that the fight to end Duchenne requires a broad approach that combines research, advocacy, care, and education, to help us understand, treat, and ultimately find a cure.

 

Optimism

We see many reasons for hope—from improved treatments to more awareness; from the dedication of our researchers to the passion of our community; from the resilience of our families to the longer lives boys are living today.

 

Knowledge

We believe that knowledge is power—whether it’s researchers seeking life-saving treatments, health care professionals looking to give the best possible care, parents who just want their child to live longer, or young men who just want to understand what’s happening to them.

 

Collaboration

We are strongest when we are a united community that works together to save lives.

 

Determination

We will not end our fight until every young man with Duchenne survives his diagnosis.