Programs

  • Program 1 [2020]

    RESEARCHPPMD'S RESEARCH PROGRAM IDENTIFIES AND AGGRESSIVELY FUNDS THE MOST PROMISING NEAR AND LONG-TERM DUCHENNE RESEARCH AND CUTTING-EDGE THERAPIES; STIMULATES NEW RESEARCH TO ENSURE THAT THE THERAPEUTIC PIPELINE IS RICH WITH OPPORTUNITY; FACILITATES PRE-COMPETITIVE INDUSTRY INTERACTIONS THROUGH THE DUCHENNE DRUG DEVELOPMENT ROUNDTABLE TO ENHANCE AND HARMONIZE THE CLINICAL TRIAL LANDSCAPE; AND SUPPORTS MULTI-STAKEHOLDER DATA MODELLING CONSORTIA TO ACCELERATE DRUG DEVELOPMENT TIMELINES. IN ADDITION TO SIGNIFICANT PRE-CLINICAL AND ACADEMIC RESEARCH FUNDING, PPMD MAKES INVESTMENTS IN EARLY-STAGE BIOPHARMACEUTICAL COMPANIES TO CATALYZE DEVELOPMENT OF NOVEL THERAPIES TO TREAT DUCHENNE AND BECKER. THE DUCHENNE REGISTRYTHE DUCHENNE REGISTRY IS A PATIENT-REPORTED REGISTRY FOR INDIVIDUALS WITH DUCHENNE AND BECKER MUSCULAR DYSTROPHY AND CARRIER FEMALES. ESTABLISHED BY PARENT PROJECT FOR MUSCULAR DYSTROPHY RESEARCH IN 2007, THE REGISTRY WAS CREATED TO BRIDGE THE INFORMATION GAP BETWEEN CLINICIANS, RESEARCHERS AND THE PATIENT COMMUNITY, THEREBY ADDRESSING MEDICAL CARE NEEDS AND ACCELERATING THE PACE OF THERAPEUTIC ADVANCEMENTS. OVER 5,000 FAMILIES ACROSS THE GLOBE HAVE EMBRACED THEIR ROLE AS CITIZEN SCIENTISTS AND HAVE CONTRIBUTED TO THE LARGEST PATIENT-REPORTED DATA SET FOR DUCHENNE.CAREPPMD'S CARE PROGRAM STRIVES TO ENSURE THAT PEOPLE LIVING WITH DUCHENNE ARE LIVING LONGER, STRONGER LIVES, BY HELPING PROVIDE ACCESS EXPERT HEALTHCARE PROVIDERS, A COMPREHENSIVE TEAM OF SUB-SPECIALISTS, AND CUTTING EDGE TREATMENTS. PPMD IDENTIFIES GAPS IN CARE FOR PEOPLE WITH DUCHENNE THROUGHOUT THE LIFESPAN AND COLLABORATES WITH HEALTH CARE PROFESSIONALS ACROSS THE GLOBE TO IMPROVE HEALTH OUTCOMES FOR ALL DUCHENNE PATIENTS. PPMD'S CERTIFIED DUCHENNE CARE CENTER PROGRAM HELPS TO ENSURE THAT CENTERS COMPLY WITH THE STANDARDS OF CARE AND SERVICES THAT HAVE BEEN ESTABLISHED IN THE DUCHENNE CARE GUIDELINES. ALL CERTIFIED DUCHENNE CARE CENTERS HAVE MET THE REQUIREMENTS FOR, AND AGREE TO PROVIDE, OPTIMAL STANDARDIZED CARE AND SERVICES.
    Geographies
    Not indicated
    Dates
    Jan 1, 2020 – Dec 31, 2020
    Source
    990
    No causes provided
    No populations provided
    –
    $5.3M

  • Program 2 [2020]

    EDUCATIONPPMD'S EDUCATION INITIATIVES INCREASE AWARENESS AND UNDERSTANDING OF THE DISEASE PROGRESSION AND THE COMPONENTS OF MULTIDISCIPLINARY COMPREHENSIVE CARE NECESSARY TO OPTIMIZE QUALITY AND QUANTITY OF LIFE THROUGHOUT THE LIFESPAN. PPMD PARTNERS WITH EXPERTS IN DUCHENNE TO DEVELOP UP-TO-DATE INFORMATIONAL RESOURCES REGARDING TREATMENT AND CARE THAT WE THEN SHARE BROADLY. PPMD COLLABORATES WITH NATIONAL AND INTERNATIONAL ORGANIZATIONS TO DEVELOP AND DISSEMINATE EDUCATIONAL RESOURCES APPROPRIATE FOR THE GLOBAL DUCHENNE COMMUNITY.
    Geographies
    Not indicated
    Dates
    Jan 1, 2020 – Dec 31, 2020
    Source
    990
    No causes provided
    No populations provided
    –
    $873.2K

  • Program 3 [2020]

    ADVOCACYPPMD'S ADVOCACY EFFORTS ARE FOCUSED ON ADVANCING CARE AND TREATMENTS FOR DUCHENNE BY LEVERAGING FEDERAL RESOURCES, BUILDING PARTNERSHIPS, AND CREATING REGULATORY PROCEDURES AND INFRASTRUCTURE. WE EMPOWER ADVOCATES WITH THE TOOLS AND INFORMATION TO ENGAGE WITH CONGRESSIONAL REPRESENTATIVES TO FOSTER DUCHENNE CHAMPIONS WITHIN CONGRESS AND TO ADVANCE LEGISLATION AND REGULATORY POLICIES, ENSURING THAT DUCHENNE PRIORITIES ARE REFLECTED ACROSS ALL GOVERNMENT AGENCIES.
    Geographies
    Not indicated
    Dates
    Jan 1, 2020 – Dec 31, 2020
    Source
    990
    No causes provided
    No populations provided
    –
    $364K