Cure MSD

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Cure MSD

Our mission is simple, yet powerful: To cure Multiple Sulfatase Deficiency. It is fatal - children with MSD rarely live past their 10th birthday.

Multiple Sulfatase Deficiency (MSD) is a rare, genetic condition that affects children. It is a monstrous disease that is extremely progressive, similar in effect to Alzheimer's or Parkinson's. Children born with MSD suffer from loss of motor skills, speech and cognitive abilities, inability to swallow, blindness and ultimately death. While this disease is rare, with only approximately 100 known c

ases throughout the world, there is hope. Since United MSD Foundation's creation in 2016, advancements in gene therapy research have been made as we aggressively move closer to a cure. Through relentless pursuit of treatments, we aspire for all children with MSD to live a healthy life without limitations. Learn more at www.CureMSD.org.

05/06/2026

Happy 9th Birthday to João Lucas! 🎉💙 Today, we are honored to celebrate you, and wish you a future filled with many more precious milestones!

João is a little boy whose bright smile, joyful spirit, and quiet strength have touched so many lives. As his mother, Dri, beautifully shares, "What really matters is that you're here... the sparkle in your eyes when you're happy... every achievement, every challenge overcome, every moment we've shared."

Every birthday is a powerful reminder of what matters most. Although he is nonverbal, João Lucas teaches us what love truly looks like, adds Dri: strength, patience, faith, and unconditional love. And if she could go back in time, she reflects, she would choose him again "a thousand times."

"My greatest wish on this birthday is ... that you continue to have health, comfort, love, and happiness. ...And may I have the privilege of continuing to be by your side, holding your hand, understanding your gaze, and celebrating every moment of your life. Happy 9th birthday, my son. You are the purest, truest, and most beautiful love I have ever known."

With love from all of us at the United MSD Foundation, we share Dri's wish for João Lucas and for every family affected by MSD: a future filled with more milestones, more reasons to celebrate, and more precious time together.

03/06/2026

📅 SAVE THE DATE: MSD WORLD DAY — JULY 30 🌎

This MSD World Day, our global community will come together to honor the strength of individuals and families affected by Multiple Sulfatase Deficiency and support the research bringing us closer than ever to potential treatments.

After years of perseverance, collaboration, and scientific progress built on hope, we are now bound for breakthroughs.

💙 Mark your calendar.
💙 Share the date.
💙 Join us July 30 as we unite around the world to propel real progress and build a brighter future for everyone battling MSD.

25/05/2026

This Memorial Day, we pause to honor the brave men and women who gave their lives in service to our country.

Their sacrifice is the foundation of the freedoms we hold today. We are forever grateful.

Wishing our U.S. community a day of remembrance, reflection, and time with those you love.

22/05/2026

We’re thrilled to welcome 🧬 Dr. Lars Schlotawa, MD, from the University Medical Centre Goettingen (UMG) to the speaker line-up for our 2026 MSD Virtual Summit!

During the Scientific Day on Sept. 18, Dr. Schlotawa will share research insights exploring drug repurposing strategies and their implications for treating MSD patients. His work reflects the growing global effort to deepen understanding of MSD and move toward more targeted therapeutic strategies for MSD families.

🗓 September 18–19
💻 ZOOM | FREE to attend
Learn more + register 👉 https://curemsd.org/2026-virtual-summit/

21/05/2026

Our 2025 Annual Report is here. 💙

Because of this community, 2025 was a year of meaningful momentum for the MSD community: advancing research, expanding family support, growing awareness, and strengthening connections around the world. Every milestone in this report reflects the resilience of MSD families, the dedication of researchers and clinicians, and the generosity of supporters who believe in a future with brighter outcomes for those living with MSD.

We’re proud to share the we made together, and filled with for what lies ahead.👇 Read the full report here:
https://heyzine.com/flip-book/d185e04d7c.html

18/05/2026

Tomorrow, May 19th, Student Ambassador Carly Sauter will take over our Instagram account to share a glimpse into her day as a future genetic counselor and UMSDF Student Ambassador!

Carly currently works as a genetic counseling assistant at Cleveland Clinic and is passionate about empathetic patient care, health equity, and raising awareness for the rare disease community.

Follow us at CureMSD on IG for stories, reels, and posts from Carly as she shares her experiences, journey, and advocacy for families impacted by MSD!

14/05/2026

More inspiring voices joining our 2026 MSD Virtual Summit speaker lineup 👇

We’re honored to welcome leading researchers advancing the future of MSD treatment and understanding through innovative pharmacological chaperone research:

🧬 Dr. Matt Baud & Dr. Hugh Britton from the University of Southampton
🧬 Dr. Hartmut Niemann from Universität Bielefeld

During our Scientific Day, these experts will share the latest developments in pharmacological chaperones and their potential role in treating MSD, including efforts focused on stabilizing the FGE protein as a therapeutic strategy.

We’re also grateful to recognize the important support of the MSD Action Foundation and the Health Research Board (HRB) Ireland, whose funding has helped advance this promising area of research.

Together, these collaborations reflect the power of global partnership, innovation, and relentless hope for the MSD community. More speakers and details coming soon!

🗓 September 18–19
💻 ZOOM | FREE to attend

Learn more here 👉 https://curemsd.org/2026-virtual-summit/

13/05/2026

UMSDF Executive Director Sarah Cortell Vandersypen is attending the Annual Meeting this week, connecting with leaders across the gene and cell therapy community and advocating for continued progress for rare diseases like .

Day 1 featured American Society of Gene & Cell Therapy's opening general session, including recognition of this year’s Catalyst Award recipients: MSD researcher Dr. Rebecca Ahrens-Nicklas, MD, Ph.D., of Children's Hospital of Philadelphia (CHOP); along with Dr. Kiran Musunuru, MD, Ph.D., MPH, ML, MRA; Dr. Fyodor Urnov, Ph.D.; and Danaher Corporation, all of whose work is helping shape the future of individualized gene therapy.

Today's sessions included the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) session, “Working as a Team to Understand the Patient Perspective,” alongside Dr. Laura Adang, MD, Ph.D., of CHOP, Melissa and Holden Duchon of Strengthan (and parents of MSD patient Ethan Duchon); Dr. Keith Gottlieb, PhD; Jocelyn Duff, MHP; and Dr. Sarah Dunsmore, MD. The session highlighted the importance of collaboration between researchers, clinicians, advocates, patients, and families in advancing AAV gene therapy development for rare diseases.

In addition to the featured panelists pictured, group photos include UMSDF Executive Director Sarah Cortell Vandersypen alongside CHOP's Dr. Laura Adang and Dr. Rebecca Ahrens-Nicklas, Melissa and Holden Duchon, and Dr. P.J. Brooks, Ph.D., and Dr. Sarah Dunsmore, Ph.D., both from NIH NCATS.

We’re grateful to see the Multiple Sulfatase Deficiency community represented in these critical conversations driving innovation, partnership, and hope forward. 💙

10/05/2026

This Mother’s Day, we honor and celebrate mothers everywhere for their boundless love, strength, and unwavering dedication that often goes unseen.

We hold deep gratitude for all mothers—especially those navigating the daily challenges of caring for a child with a rare disease, carrying that responsibility with quiet resilience and extraordinary courage.

We also hold close the mothers missing their children today, and all those carrying the ache of missing their moms. For many, this day is filled with both love and grief, remembrance and longing.

Today, and every day, you are seen, honored, and deeply appreciated. 💙

08/05/2026

Happy 6-year work anniversary, Brenna! 🎉

Your compassion, expertise, and unwavering dedication as both our Patient & Family Advocate and beloved genetic counselor have made such a meaningful difference in the lives of so many MSD families.

Thank you, Brenna, for always showing up with heart, empathy, and a deep commitment to every person you support. Our community is stronger because of you. 💖

We’re so grateful to have you as part of the United MSD Foundation family. Here’s to all you’ve accomplished and all the lives you’ll continue to touch!

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