@parentprojectmd

parentprojectmd has been a member of Linktree for 7 years and joined in February 2019. The social media accounts linked to from parentprojectmd are: • Facebook • Instagram • YouTube • LinkedIn • Email • X Besides social media accounts, parentprojectmd has populated their site with: • Highlights from PPMD's 2026 Annual Conference - Parent Project Muscular Dystrophy • Webinar: Your Voice Matters - Preparing for the FDA Advisory Committee Meeting on Deramiocel - Parent Project Muscular Dystrophy • FDA Accepts sNDAs to Review Traditional Approval for AMONDYS 45 and VYONDYS 53 - Parent Project Muscular Dystrophy • PPMD's 2027 Annual Conference • Catching up on What’s Happening at PPMD’S 2026 Annual Conference - Parent Project Muscular Dystrophy • Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44 • Solid Biosciences Shares Initial Data from INSPIRE DUCHENNE Study of Gene Therapy Candidate SGT-003 • PPMD's 2025 Annual Conference | June 19-21, 2025 | Las Vegas, NV • Avidity Biosciences Announces Plans to File Biologics License Application for Delpacibart Zotadirsen (AOC 1044) • Thank You for Joining us to Advance the Research Evolution • Webinar: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy • FDA Announces Advisory Committee Meeting to Review Capricor Therapeutics’ Biologics License Application for Deramiocel - Parent Project Muscular Dystrophy • Webinar Recording: Solid Biosciences — Update on Gene Therapy Candidate SGT-003 • Action Alert: Ask Congress to Protect NIH Research Funding for Duchenne • PPMD Lighthouse Workshop Series 2025 • Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel • Solid Biosciences Shares Updates on SGT-003 - Parent Project Muscular Dystrophy • Duchenne Newborn Screening: Next Steps Following ACHDNC Dissolution • Capricor Therapeutics Shares Positive Five-Year HOPE-2 OLE Data for Deramiocel in Duchenne - Parent Project Muscular Dystrophy • ACTION ALERT: Help Advance Duchenne and Becker Research • Congress Moves Forward on Medicaid Cuts: What You Need to Know • Cumberland Pharmaceuticals Announces Positive Results from Phase 2 FIGHT DMD Trial of Ifetroban for Treatment of Duchenne Cardiomyopathy • Together, let’s advance the research evolution • Life is about time and timing when living with Duchenne • Support PPMD’s commitment to moving the science further, faster • REGENXBIO Initiates Pivotal Phase of AFFINITY DUCHENNE® Trial of RGX-202 Gene Therapy, Reports Positive Functional Data • Inside the HHS Clinical Trials Roundtable: Why Speed, Access, and Rigor Matter for the Duchenne Community - Parent Project Muscular Dystrophy • Your gift to PPMD is a commitment to accelerating progress • Honor Every Journey - Father's Day 2026 • WATCH: Sevasemten - CANYON Trial Topline Results (Webinar Recording) • Percheron Announces Termination of Phase 2B Study of Avicursen for Duchenne • PepGen Announces Clinical Hold in the U.S. on IND Application to Initiate CONNECT2-EDO51 Phase 2 Study of PGN-EDO51 • Edgewise Therapeutics Announces Positive Topline Results from CANYON Phase 2 Trial of Sevasemten in Individuals with Becker • WATCH: RGX-202 - AFFINITY DUCHENNE Pivotal Program and Functional Data (Webinar Recording) • REGENXBIO Shares Updates on Phase I/II AFFINITY DUCHENNE® trial of RGX-202 • Solid Biosciences Shares Updates on INSPIRE DUCHENNE Clinical Trial of SGT-003 • Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne • Massachusetts Begins Newborn Screening for Duchenne - Parent Project Muscular Dystrophy • Dr. Peter Marks Resigns from FDA Center for Biologics Evaluation and Research • Duchenne Secures $12.5 Million in CDMRP Funding for FY25: A Hard-Fought Victory in a Challenging Year • PPMD's Living Duchenne Podcast: Season 7 • Webinar: RGX-202 - AFFINITY DUCHENNE Pivotal Program and Functional Data • PPMD Joins FDA Leadership for Rare Disease Roundtable - Parent Project Muscular Dystrophy • The Impact of the Continuing Resolution and Federal Budget Cuts to the CDMRP on Duchenne Research • URGENT Action Alert: Urge the Senate to Protect Duchenne Research Funding in the Continuing Resolution • Action Alert! Urge Congress to Support Progress in the Fight to End Duchenne • Advocates Unite in Washington D.C. to Champion Duchenne Policy Reforms • Dyne Submits BLA to FDA for z-rostudirsen (DYNE-251) in Duchenne Amenable to Exon 51 Skipping - Parent Project Muscular Dystrophy • Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS • 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 • 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 • Webinar: Sarepta Therapeutics — Safety Update on ELEVIDYS in Non-Ambulatory Individuals Living with Duchenne • 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 • 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) • Webinar: Wave Life Sciences - Update on FORWARD-53 Study of WVE-N531 • 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 • Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit • 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 • PPMD Convenes GI, GU, and Swallowing Experts for Two-Day Workshop • 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 • 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 • 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 • Support PPMD’s Gene Therapy Initiative with Your Gift • 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 • Thank You for Fighting for Every Future With Us • 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 • 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 • 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 • 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 • >> What is Duchenne? • >> The Duchenne Registry • >> Upcoming Events • >> PPMD Website • >> DONATE