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Massachusetts Begins Newborn Screening for Duchenne - Parent Project Muscular Dystrophy

PPMD's Living Duchenne Podcast: Season 7

PPMD Joins FDA Leadership for Rare Disease Roundtable - Parent Project Muscular Dystrophy

Dyne Submits BLA to FDA for z-rostudirsen (DYNE-251) in Duchenne Amenable to Exon 51 Skipping - Parent Project Muscular Dystrophy

Dyne Announces Phase 3 Trial of Z-Rostudirsen, Plans for Submitting Biologics License Application to FDA

PPMD's 2026 Annual Conference

Donate to PPMD’s Adult Care Initiative 2026

GEMMABio Announces Duchenne Muscular Dystrophy Program, Shares Preclinical Data for Investigational Gene Therapy for Duchenne

REGENXBIO Shares Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202

Donate to Honor Every Journey - Mother's Day 2026

Momentum Continues: Duchenne Newborn Screening Advances Across the US

Advancing Duchenne Newborn Screening: A Collaborative Path Forward

Webinar: Exercise and Dystrophinopathy - What Families Need to Know - Parent Project Muscular Dystrophy

Treatment Preference Study

Webinar: Ataluren and the Current Regulatory Landscape

PTC Therapeutics Shares Updated Community Letter Regarding Ataluren - Parent Project Muscular Dystrophy

PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy - Parent Project Muscular Dystrophy

Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53

Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne

PPMD Together: Philadelphia | April 24-25, 2026

Community Comes Together on Capitol Hill: A Recap of PPMD’s 2026 Advocacy Conference

ACTION ALERT: Urge Congress to Support Progress in the Fight to End Duchenne and Becker

Capricor Therapeutics Announces New PDUFA Date for Deramiocel

PPMD and FED Announce 2026 Advocacy Leadership Awards Honoring Bipartisan Champions in Congress - Parent Project Muscular Dystrophy

PPMD Partners with Cooperative International Neuromuscular Research Group, Global Sponsors to Advance Expanded Duchenne Natural History Study - Parent Project Muscular Dystrophy

25 Years Later: Reflecting on the 2001 MD-CARE Act Hearing and Where We Are Now

PPMD For You

Cardiac Care for Female Carriers

PPMD Tween Virtual Social | Feb. 10, 2026

PPMD Lighthouse Workshop Series 2026

PPMD Joins Collaborative Research Effort to Decode How Human Muscle Regenerates

Sarepta Shares Topline Three-Year EMBARK Data for ELEVIDYS in Ambulatory Duchenne Patients

ACTION ALERT: Urge Congress to Support FY26 Duchenne Funding

Webinar: Dyne Therapeutics — Topline Results from Phase 1/2 DELIVER Trial

Newly Diagnosed Meet & Greet | Jan. 14, 2026

PPMD’s Race to End Duchenne Team Member Claims Victories in Both 5K and 10K as Community Raises Funds for Duchenne and Becker Research and Care

PPMD's 2026 Advocacy Conference | March 8-10

Honor Every Journey with PPMD

Honoring Every Journey in Duchenne and Becker: Advancing Therapies, Access, and Care For All - Parent Project Muscular Dystrophy

Webinar: Capricor Therapeutics — HOPE-3 Community Update

Historic Victory: Duchenne Added to the Recommended Uniform Screening Panel (RUSP)

U.S. Department of Health and Human Services Adds Duchenne to Recommended Uniform Screening Panel

Webinar: Gene Therapy for Duchenne - Translating clinical guidance into meaningful patient understanding

Capricor Therapeutics Announces Positive Topline Results from HOPE-3 Study of Deramiocel - Parent Project Muscular Dystrophy

Approved Therapies for Duchenne

A Thanksgiving Message from Pat

Watch the Recording: Avidity Biosciences - EXPLORE44-OLE® Topline and Functional Data Presentation

PPMD Launches New Study to Understand Patient and Caregiver Treatment Preferences

Avidity Biosciences Announces U.S. Managed Access Program for del-zota

FDA Approves New Safety Warning and Revised Indication for ELEVIDYS

Webinar: Avidity Biosciences - EXPLORE44-OLE® Topline and Functional Data Presentation

Welcoming the New Members of PAAC for Fall 2025

Pat Chat: Get to Know PPMD CEO Katherine Beaverson, MS

Solid Biosciences Shares Interim Data from INSPIRE DUCHENNE Trial of SGT-003

PPMD Provides $400,000 in Funding to MyoGene Bio Through PPMD Venture Pathways Program to Support Development of Gene Editing Platform - Parent Project Muscular Dystrophy

Sarepta Announces Completion of ESSENCE Trial for Exon 45 & 53 Skipping Therapies - Parent Project Muscular Dystrophy

Becker Muscular Dystrophy Webinar Series – Part 2: Cardiopulmonary Care in Becker Muscular Dystrophy - Parent Project Muscular Dystrophy

Italfarmaco Addresses Patient Deaths, DUVYZAT Safety Profile in Community Letter - Parent Project Muscular Dystrophy

Webinar: Staying Safe - Avoiding Falls and Recognizing Signs of Fat Embolism Syndrome (FES) - Parent Project Muscular Dystrophy

PPMD Together: Virtual | November 17

20th Annual Sam’s Night Event Marks Two Decades of Community Impact, Growth, and Hope for Families Living with Duchenne - Parent Project Muscular Dystrophy

Newly Published Study Evaluates Patients’ and Caregivers’ Acceptable Risk from Gene Therapy - Parent Project Muscular Dystrophy

Tween Virtual Social | October 21, 2025

Avidity Biosciences Announces Positive Pre-BLA Meeting with FDA for del-zota - Parent Project Muscular Dystrophy

Standing Together for Access: Ensuring Duchenne Patients Can Benefit from Approved Therapies - Parent Project Muscular Dystrophy

Catch Up on World Muscle Society 2025

Building Protection: Vaccines and Dystrophinopathy

Webinar: Neuromuscular Care and Genetic Considerations in Becker - Parent Project Muscular Dystrophy

PJ Nicholoff Steroid Protocol

Catch Up on PPMD Together: Charlotte

PPMD's Sibs Connect

Ready for Respiratory Season: Keeping You and Your Family Healthy - Parent Project Muscular Dystrophy

Webinar: Building Protection - Vaccines and Dystrophinopathy - Parent Project Muscular Dystrophy

Get Involved: Fundraise for PPMD

Duchenne on the World Stage (by Patrick Moeschen)

Avidity Biosciences Announces Positive New Data from EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 Studies - Parent Project Muscular Dystrophy

Bad Shirt Friday 2025 - Make a Donation

Understanding BiPAP Webinar: Why It’s Needed, What Gets in the Way, and, How to Increase Compliance - Parent Project Muscular Dystrophy

World Duchenne Awareness Day 2025

#BadShirtFriday | September 5, 2025

PPMD Together: Charlotte

Becker Education & Engagement Day 2025

Newly Published Consensus Guidelines Aim to Ensure Safe and Equitable Delivery of Gene Therapy in Duchenne - Parent Project Muscular Dystrophy

Understanding BiPAP: Why It’s Needed, What Gets in the Way, and How to Increase Compliance

Expanding Leadership, Accelerating Progress

Tween Social - 8/21/25

Make Your Voice Heard: Support Adding Duchenne to the RUSP

PPMD Lighthouse Workshops

Get in the Game with Coach to Cure MD

Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population

Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population

Webinar: Capricor Therapeutics - Regulatory Update and Clinical Insights on Deramiocel for Duchenne Cardiomyopathy

FDA Grants Breakthrough Therapy Designation to Avidity’s del-zota

Update on ELEVIDYS Shipments in the U.S.

Post AAV Gene Therapy Resources

Capricor Therapeutics Receives Complete Response Letter Regarding Deramiocel (CAP-1002)

2025 Annual Conference Recordings

Medicaid, Reconciliation Passage, and What Comes Next for Our Community

Webinar Recording: Edgewise Therapeutics - Update on Sevasemten Program in Becker and Duchenne

ACTION ALERT: Tell the Senate – Protect Medicaid and the Families Who Rely On It

A Safety Update on ELEVIDYS - June 2025

PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation

Inclusive Summer Activities

PPMD's Certified Care Center Program

Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol

Webinar: Dyne Therapeutics — The Clinical Impact of the FORCE Platform for Duchenne

Satellos Announces Positive Functional Data from Phase 1b Open-Label Trial of SAT-3247 in Adults with Duchenne

Medicaid Impact

Webinar Recording: Discover the Power of Your Data - The Duchenne Registry

Florida Signs Duchenne Newborn Screening into Law

A Decade of Impact: Honoring PJ’s Protocol and Its Legacy

ACTION ALERT: Help Advance Duchenne and Becker Research

Budget Reconciliation Update and the Impact on Medicaid

ACTION ALERT: Protect Medicaid

Share your NOW: The impact of up-to-date data in The Duchenne Registry

Recording: Sarepta Therapeutics - Duchenne Gene Therapy Update

Recording: Wave Life Sciences - Update on FORWARD-53 Study of WVE-N531

Watch: Avidity Biosciences - Topline Data from EXPLORE44® Clinical Trial (Webinar Recording)

Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial

REGENXBIO Shares Positive Biomarker Data from AFFINITY DUCHENNE Trial of RGX-202 Gene Therapy

Webinar: Avidity Biosciences – Presentation and Discussion on Topline Data from EXPLORE44® Clinical Trial

A Safety Update on ELEVIDYS

Avidity Biosciences Announces Positive Topline Del-zota Data in Phase 1/2 EXPLORE44® Trial

Webinar: Capricor Therapeutics - Update on Deramiocel (CAP-1002) for the Treatment of Cardiomyopathy in Patients with Duchenne

Capricor Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application for Deramiocel

PPMD's Knight Hacks

Advance the Research Evolution with The Duchenne Registry

PPMD Advocacy in the 119th Congress and the 47th Administration

PPMD's Race to End Duchenne Program Celebrates 20 Years of Going the Distance to End Duchenne

WATCH: Exploring Muscle Regeneration - A New Approach with Satellos (Webinar Recording)

Webinar: INSPIRE DUCHENNE Clinical Trial Update and Study Expansion

FDA Accepts Translarna NDA Resubmission for Review

Flu Season Ready: Protecting Yourself and Your Family

PPMD Together: Virtual

Webinar: Exploring Muscle Regeneration A New Approach with Satellos

Catch Up on World Muscle Society 2024

WATCH: SHIELD DMD - A Bone Health Study in Duchenne (Webinar Recording)

Emergency Preparedness for People with Disabilities

THINK Resources

Expert Care in an Emergency: Important Contact Information & Resources

EHR Study

PPMD: 30 Years of Progress

WATCH: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy (Webinar Recording)

Webinar: SHIELD DMD: A Bone Health Study in Duchenne

Action Alert! Urge Your Senators to Support the Creating Hope Reauthorization Act

PPMD Hosts Meeting Exploring the Case for Cardiac Transplantation in Duchenne

The Duchenne Registry

PPMD Joins Duchenne Parent Project aps Respiratory International Professional Care Meeting

Fundraise to End Duchenne

Catch Up on PPMD Together: Seattle

Thanks to You PPMD Continues Forward in All Areas of Support

Capricor Therapeutics Plans to File Biologics License Application for Full Approval of Deramiocel for the Treatment of Duchenne Cardiomyopathy

Coach to Cure MD 2024

Wave Life Sciences Announces Positive Interim Data from FORWARD-53 Clinical Trial Evaluating WVE-N531 in Individuals With Duchenne Amenable to Exon 53 Skipping

Race to End Duchenne Virtual Series - Racing For Every Future

DIY Care Binder | Order Support Materials

PPMD Contributes to National Academies Report on Rare Disease Regulatory Processes

PPMD Community Webinar Recap: Overview of DUVYZAT and Access Resources

PPMD & Global Patient Advocacy Groups Award BIND Project $113,000 to Further Brain Involvement Research

Navigating Access & Resources

Watch the Recording: Advocating for A Successful School Year

PPMD Lighthouse Workshop Series 2024

Community Webinar: Overview of DUVYZAT and Access Resources

WATCH: PPMD Advocacy - Raising Our Voices in 2024 and Beyond

Introducing PPMD’s Newborn Screening Action Center: A Hub for Advocacy and Education

Duchenne Action Month 2024

Webinar: Advocating for a Successful School Year

Dyne Therapeutics Announces Positive New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne

WATCH: Advancing DYNE-251 - An Investigational Medicine for Duchenne to Deliver for Patients (Webinar Recording)

Becker Education & Engagement Day 2024

Finding Support to Manage Behaviors in Duchenne and Becker

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA

WATCH: Edgewise Therapeutics – Clinical Trials in Becker Muscular Dystrophy (Webinar)

Avidity Biosciences Announces Positive Data From Phase 1/2 EXPLORE44™ Trial of AOC 1044 in Individuals Amenable to Exon 44 Skipping

Your Citizen Science At Work

Webinar: Advancing DYNE-251 - An Investigational Medicine for Duchenne to Deliver for Patients

PPMD's Living Duchenne Podcast: Season 6

PPMD For You

Clinical Trials & Outcome Measures in Duchenne

Webinar: Edgewise Therapeutics - Clinical Trials in Becker Muscular Dystrophy

Finding Belonging and Fostering Community

PepGen Announces Positive Data from Phase 2 Clinical Trial of PGN-EDO51

Pfizer Discontinues Development of Investigational Mini-Dystrophin Gene Therapy

PPMD Responds to July 29th STAT Article

Making it Work: Practical Tips for Enhancing Quality of Life

ITF Therapeutics Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Duchenne

Navigating the Approved Therapy Landscape

PPMD Applauds FDA Launch of Rare Disease Innovation Hub — A Milestone for Rare Disease Patients

PPMD's Adult Advisory Committee (PAAC)

PPMD's 2025 Annual Conference | June 19-21, 2025 | Las Vegas

Capricor Therapeutics Announces Positive Skeletal Muscle and Cardiac Function Data from HOPE-2 OLE Study Data of Deramiocel (CAP-1002)

PPMD Convenes Leading Experts to Discuss Topics in Becker Care Ahead of 30th Annual Conference

Find a Certified Duchenne Care Center

PPMD's 30th Annual Conference | June 27-29, 2024 | Orlando, FL

FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older

PAAC Travel Tips

Newborn Screening for Duchenne: PPMD's Collaborative Future Planning Efforts

Cranbury Pharmaceuticals (Tris Pharma) Receives FDA Approval for First Generic Version of Emflaza® (deflazacort) for Duchenne

Build Your Own Care Binder at PPMD’s 30th Annual Conference

Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy

Consensus Statement Featuring Expert Insights from PPMD’s Certified Duchenne Care Centers on Access to Novel Therapies for Duchenne Published

Roche Announces Phase II Open-Label SHIELD DMD Study to Assess Effect of Satralizumab on Bone Health in Duchenne

Summer Fun: Accessible Activities for All Ages and Interests

Find a Local PPMD Connect Group

Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002

NS Pharma Shares Update on VILTEPSO® (Viltolarsen) Phase 3 Study

Dyne Therapeutics Shares New Clinical Data from DELIVER Trial of DYNE-251 in Duchenne Patients

Study to Explore Barriers to Diverse Clinical Trial Participation in Duchenne Published

PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation

Mapping Out a Path Forward: PPMD Convenes Representatives from 21 Academic Centers and 11 Industry Partners for 2024 Cardiac Care Workshop

Give Monthly to PPMD

Congress Passes FAA Reauthorization Featuring Monumental Accessibility Improvements

PPMD’s Pat Furlong Honored with ASGCT’s Sonia Skarlatos Public Service Award

Updates from the ACHDNC Meeting and Next Steps for Duchenne Newborn Screening

PPMD Hosts 2024 Cardiac Workshop, May 9-10

Update on Pfizer’s Phase 2 Gene Therapy Trial for Duchenne

WATCH: Community Progress in Duchenne Newborn Screening (Webinar Recording)

Comprehensive Genetic Counseling Resource for Dystrophinopathies Published: A Collaborative Effort by PPMD and Expert Genetic Counselors

Welcoming Jodi Wolff, PhD: A Passionate Advocate Joins PPMD’s Team as Chief Program Officer

Thank You for Honoring PPMD’s 30th Anniversary and Fighting for Every Future With Us

Action Alert! Urge Your Senators to Support Progress in the Fight to End Duchenne

Catch Up on PPMD’s Inaugural PPMD Together Event in Cincinnati, Ohio

PPMD Awards $250,000 to Support Clinical Research Network for Duchenne Babies Identified by Newborn Screening

Webinar: Community Update on Duchenne Newborn Screening

Duchenne UK and PPMD Award $500,000 to Evaluate Safety, Tolerability of Muscle Progenitor Cells in Phase 1 Trial

Ohio to Begin Screening Newborns for Duchenne

PPMD Celebrates 30th Anniversary with Launch of PPMD Together Meeting Series in Cincinnati

Edgewise Therapeutics Announces Positive Two-Year Topline Results from ARCH Study of EDG-5506

Duchenne Siblings Study Published

PPMD Launches First State-Specific Duchenne Advocacy Day in Illinois

60+ Experts Unite to Author Consensus Statement Championing Duchenne Newborn Screening

Dads Monthly Meeting - April | Virtual

WATCH: Community Update with ITF Therapeutics – Introducing DUVYZAT™ (givinostat) (Webinar Recording)

Fiscal Year 2024 Appropriations Package Signed Into Law, Including Critical Funding for Duchenne and Becker

Community Update with ITF Therapeutics: Introducing DUVYZAT™ (givinostat)

ITF Therapeutics Shares Update on DUVYZAT™ Following FDA Approval

FDA Grants Approval to DUVYZAT™ (Givinostat)

WATCH: Sarepta Therapeutics – Data update from MOMENTUM study, SRP-5051 (Webinar Recording)

PPMD’s 2024 Advocacy Conference Brings Together Community to Support Progress in Fighting for Every Future

PTC Therapeutics Announces Plans to Resubmit Translarna™ NDA to FDA

WATCH: NS Pharma - Overview of New Exon Skipping Trials: Brogidirsen and NS-050/NCNP-03 (Webinar Recording)

The Duchenne Registry: New Gene Therapy Survey

Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne

Care Consideration for Duchenne

Webinar: NS Pharma — Overview of New Exon Skipping Trials: Brogidirsen and NS-050/NCNP-02

Action Alert! Urge Congress to Support Progress in the Fight to End Duchenne

PPMD Hosts Town Hall on Clinical Trials During 2024 Advocacy Conference

Meet the 2024 PPMD Adult Advisory Committee (PAAC) Members

Support PPMD Advocates on Rare Disease Day - Sign up for Action Alerts

PPMD Together: Cincinnati

Breaking news: Rare Disease Day Congressional Hearing to Address the BENEFIT Act

Revised Community-Led Duchenne Guidance Published: Developing Potential Treatments for the Entire Spectrum of Disease

PPMD Advocacy Conference 2024

PPMD Announces Pediatric Certified Duchenne Care Center at Penn State Health Children’s Hospital

PPMD's Gene Therapy Hub

FDA Accepts Efficacy Supplement to Expand ELEVIDYS Indication

WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)

Wave Life Sciences — Overview of WVE-N531 and the FORWARD-53 Clinical Trial

PPMD Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program

Update From PPMD’s 2024 PPMD Duchenne Healthcare Professionals Summit

Watch: Dyne Therapeutics — Advancing DYNE-251, an Investigational Medicine for DMD (Webinar Recording)

REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

Decode Duchenne Program

Duchenne Newborn Screening Bill Passes Arizona Senate

PTC Therapeutics — EMFLAZA® Landscape in 2024

Watch: Navigating Steroid Choices - Understanding the Process, Pathways, and Policies (Webinar Recording)

PPMD 2023 Duchenne Early Care Meeting Report Published: Enhancing Support in Early Diagnosis

Duchenne Added to Minnesota’s Newborn Screening Panel

CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe

Dyne Therapeutics — Advancing DYNE-251, an Investigational Medicine for DMD, to Deliver for Patients

Navigating Steroid Choices: Understanding the Process, Pathways, and Policies

PPMD Hosts 2024 Duchenne Healthcare Professionals Summit

NEW! PPMD Together

The Importance of Assisted Cough

Dyne Therapeutics Announces Positive Initial Clinical Data from DELIVER Trial in Duchenne Patients

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label

PPMD & Community Partners Urge Timely, Transparent FDA Review of EMBARK Data

RSV, COVID, and Flu - oh my!

Wave Life Sciences Announces Initiation of Dosing in Phase 2 Duchenne Clinical Trial of WVE-N531

Watch: Community Update on the New INSPIRE DUCHENNE Clinical Study of SGT-003 (Webinar Recording)

PTC Therapeutics Shares Update on Translarna™ Regulatory Activities

Watch: AGAMREE® Approval with Catalyst Pharmaceuticals, Inc. (Webinar Recording)

Empowering Carriers of Duchenne: New Carrier Publications Resource

Webinar: Community Update on the New INSPIRE DUCHENNE Clinical Study of SGT-003

Italfarmaco Announces PDUFA Extension for the FDA's Review of Givinostat

CDCC Spotlight: Children's Hospital of Richmond at VCU

Solid Biosciences Receives FDA Clearance for Investigational New Drug Application for SGT-003 Gene Therapy Candidate

Advancing Duchenne Cardiac Care: A Year of Impact and Gratitude

PPMD's 15 Year Registry Report

Webinar: AGAMREE® Approval with Catalyst Pharmaceuticals, Inc.

PPMD Provides $250,000 in Funding to Rare Disease Research to Establish New Clinical Trial Site

WATCH: AGAMREE Approval - What this means for you & your family (Webinar recording)

Webinar: Overview of Topline Results from Phase 3 EMBARK Study

Defeat Duchenne Canada and PPMD Award $300,000 (USD) Clinical Fellowship in Duchenne Endocrinology and Bone Fragility

Common Labs Resource

WEBINAR: AGAMREE® Approval What this means for you and your family

Sarepta Therapeutics Announces Topline Results from Phase 3 EMBARK Study of ELEVIDYS Gene Therapy

FDA Grants Approval to AGAMREE® (Vamorolone)

Duchenne Added to New York State's Newborn Screening Panel

Community Report: Key Insights from the April 2023 Endocrine and Bone Workshop

Milestone in Minnesota: Duchenne Newborn Screening Heads to Commissioner's Desk

PPMD Lighthouse Workshop Series 2023

Tween Social (ages 9-14) | Virtual

PPMD's End Duchenne Tour: Chicago, IL

Webinar: Advocating for Access - Overview of Medicaid DUR and P&T Meetings

Capricor Therapeutics: CAP-1002 HOPE-3 Program Update

WATCH: Supporting Families Through Decision Making (Webinar Recording)

FibroGen announces discontinuation of LELANTOS-2 Open Label Extension

Santhera Receives Positive European Medicines Agency (EMA) CHMP Opinion Recommending Approval of Vamorolone in European Union

PPMD & Edgewise Therapeutics: A Novel Approach Designed to Protect Muscle in Becker Muscular Dystrophy

Care Checklists

Finding and Accepting Help

Webinar: Supporting Families Through Decision Making

What is Palliative Care? Understanding an additional layer of support

PPMD’s Connect Community Chats: Accepting Help | Oct 3

PPMD’s Connect Community Chats: Accepting Help | Sept 27

CDCC Spotlight: Children's Hospital Los Angeles

WEBINAR: Advocating for Access: Insurance 101

Game Day Tips for Coach to Cure MD

Duchenne Action Month: Get involved with PPMD

Duchenne Action Month with PPMD

Care Resources for Carriers

CDCC Spotlight: Arkansas Children's Hospital

FibroGen Announces Topline Data from Phase 3 LELANTOS-2 Study

WATCH: Advocating for Access: Navigating Approved Therapies in Duchenne

PPMD Awards $65,000 to Vanderbilt University Medical Center to Advance Understanding of Duchenne Cardiomyopathy

Safe & Unsafe Medications in Duchenne

CDCC Spotlight: Children's Health of Dallas

WEBINAR: Advocating for Access: Navigating Approved Therapies in Duchenne

School Resources

PPMD’s RUSP nomination package for Duchenne passes first hurdle in federal committee vote

PPMD’s Duchenne Educators Masterclass

Join our 2023 Coach to Cure MD team!

CDCC Spotlight: Children’s Hospital Colorado

New Clinical Experiences Survey

CDCC Spotlight: Nemours Children’s Hospital

2023 PPMD Annual Conference Session Recordings

Duchenne Newborn Screening milestone in Ohio

ELEVIDYS Approval: What This Means For You and Your Family

PPMD’s 2023 Annual Conference: Day 3

PPMD’s 2023 Annual Conference: Day 2

PPMD’s 2023 Annual Conference: Day 1

Register for PPMD's 2023 Annual Conference, June 29-July 1 in Dallas, Texas

Italfarmaco Group Announces NDA Accepted for Givinostat

Edgewise Therapeutics announces positive topline 12-month data from EDG-5506 ARCH study

PAAC Travel Tips

FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Santhera announces exclusive North America license agreement with Catalyst Pharmaceuticals for vamorolone

Thanks to you PPMD continues forward in all areas of support

WATCH: ENVISION Study Update With Sarepta Therapeutics (Webinar Recording)

PPMD Announces Pediatric Certified Duchenne Care Center at Norton Children’s Hospital

2023 Annual Conference App: Login Instructions

Study: Duchenne patients’ and caregivers’ perspectives on corticosteroids

PPMD Announces Pediatric Certified Duchenne Care Center at Boston Children’s Hospital

Collaborative Research Grant for DMD Announced

Over 1,000 Free Carrier Tests Performed through PPMD’s Decode Duchenne Program!

Sarepta Announces Update on ENVISION Study Enrollment in U.S.

FibroGen Announces Topline Data from Phase 3 LELANTOS-1 Study

Blog: Updated Duchenne School Resources

Race Athlete Spotlight: Tiffany Mulloy

Agenda - PPMD's 2023 Annual Conference

Update on PPMD’s newborn screening efforts

Sarepta Announces PDUFA Extension and Update for the FDA's Review of SRP-9001

Virtual Town Hall: SRP-9001 Advisory Committee Update

A Community United: Thank You for Raising Your Voice During the SRP-9001 Advisory Committee

May 12: Advisory Committee Meeting for SRP-9001 - Live Stream Information & Meeting Materials Released

PPMD Submits Findings from New Gene Therapy Preference Study to FDA for SRP-9001 Advisory Committee

End Duchenne Tour - Long Island

PPMD Knight Hacks | Everyday Tips & Tricks for Families

Race to End Duchenne Virtual Series - 79 Mile Challenge!

Leveraging Our United Voices: A Call To Collaborative Action Ahead of FDA's Advisory Committee for SRP-9001

PPMD Virtual Social Events

Action Alert! Urge Congress to Support Progress in the Fight to End Duchenne

PPMD Announces Pediatric Certified Duchenne Care Center at Phoenix Children’s

PPMD's 2023 Advocacy Conference | March 5-7 | Washington, DC

PPMD For You: Schedule a Meeting

PPMD Submits Updated Community Draft Guidance

Become a PPMD Advocate

PPMD’s Connect Program

Coach To Cure MD

Create a DIY Fundraiser

Upcoming Virtual Socials

Race to End Duchenne

The Duchenne Registry: Advancing Research for Female Carriers

PPMD's Decode Duchenne Genetic Testing Program

Duchenne MD Carriers: Your Questions Answered

PPMD's Living Duchenne Podcast: Season 4

September is #DuchenneActionMonth! Celebrate Your Actions

PPMD Merch Store

In an Emergency, Remember to T.H.I.N.K. — New PPMD Emergency Care Bundle Now Available!

PPMD Submits RUSP Nomination Package for Duchenne

Breaking News! PPMD Awards $2 Million to ACTION Network to Revolutionize Duchenne Cardiac Care

Action Alert: Support Progress in the Fight to End Duchenne

PPMD COVID-19 Information Center

Becker Muscular Dystrophy Research

PPMD's Masterclass for Educators

Teens & Adults: Join PAAC Chats

COVID-19 Vaccination & Duchenne: FAQs

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