Patient Community

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Learn more about Adam and Lea’s story

Patients Review

 

“We are inspired by Adam and Lea, siblings living with Sanfilippo Syndrome Type B (MPS IIIB).”

“Their bond reminds us that this journey affects whole families. Their strength, love, and resilience fuel our drive to push harder—because every moment matters when you’re fighting for each other.”

PATIENTS ABOVE ALL ELSE

At Spruce Bio, we know that behind every scientific breakthrough is a patient, a family, and a story that matters. Navigating any disease is deeply personal, and we are committed to being a trusted partner to the MPS IIIB community. The Spruce Patient Engagement Liaison (PEL) Program was created to ensure that you have direct access to our team, timely information, and a voice in the journey ahead.


LETTER FROM OUR CHIEF EXECUTIVE OFFICER, JAVIER SZWARCBERG, M.D., M.P.H

Dear MPS Community,

On behalf of the entire Spruce family, I want to personally connect with you as we begin this important next chapter for the Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) program, formerly known as AX 250.

We recognize that this message may reach individuals and families who have been part of this journey for some time—many of whom participated in or closely followed earlier phases of the program. We also understand that the path to this point has not always been clear, and that for some, the pause in development may have brought frustration, concern, or uncertainty.

Spruce Bio is now leading the development of TA-ERT, and I want to assure you that we are fully committed to advancing this program with the urgency, care, and transparency it deserves. Since taking over responsibility for the program, we have been focused on reinitiating manufacturing to ensure clinical drug product is available—an essential first step toward restarting clinical activities and building on the important progress already made.

Our goal is clear: to make TA-ERT available as quickly and responsibly as possible. We intend to pursue accelerated approval in the United States, while preparing to initiate a registrational clinical study and broaden global early access opportunities in early 2026. We are planning to meet with regulators and we will continue to keep you informed as we reach new milestones and progress through each phase.

We understand that trust must be earned, especially following a period of uncertainty. While this message may not answer every question, please know that we are exploring opportunities for additional communication and engagement to share more about our approach and hear directly from you.

Above all, we want to recognize the strength, resilience, and hope that define this community. Your continued perseverance inspires us every day. We are here for you, and we remain focused on what matters most: delivering TA-ERT to those who need it—safely, urgently, and with care.

If you have any questions or would like more information, we invite you to contact the Spruce Patient Engagement Liaison Team at PEL@sprucebio.com. We are here to support you.

With warmest regards,

Javier
and the entire Spruce Team

WHAT IS THE PEL PROGRAM?

The Spruce PEL Program is more than a resource—it’s a connection. Designed to support individuals and families affected by MPS IIIB and other serious diseases, our PEL team serves as a dedicated bridge between the patient community and Spruce Bio. We are here to listen, learn, and collaborate, ensuring that patient needs are at the core of everything we do.

HOW WE SUPPORT YOU

  • PERSONALIZED CONNECTION – Our PEL team serves as a dedicated point of contact to answer your questions, provide guidance, and offer tailored support throughout your unique journey.
  • EDUCATION AND RESOURCES – We provide clear, up-to-date information about MPS IIIB, clinical trials, and the latest research developments in our Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) program.
  • PATIENT AND ADVOCACY COLLABORATION – We work hand-in-hand with patient communities and advocacy organizations to ensure your experiences and perspectives shape our work.
  • ONGOING COMMUNICATION – We are committed to keeping you informed at every stage of our clinical development, listening to your feedback, and sharing timely updates.

CONNECT WITH THE PEL TEAM

If you have questions, need resources, or simply want to connect, we encourage you to reach out.
Email us at: PEL@sprucebio.com

 

PATIENT ADVOCACY

PATIENT ADVOCACY

PATIENT ADCOCACY TEXT

At Spruce Bio, we are dedicated to transforming the lives of patients living with neurological disorders. Through compassion and cutting-edge science, our efforts are built around the needs of patients and their loved ones. We listen intently to the patient communities we serve and work fiercely towards the advancement of patient services that improve access, outcomes, and quality of life.

ADVOCACY ORGANIZATIONS

We appreciate the commitment and determination lead by the Sanfilippo advocacy groups around the world and are grateful to work together to serve this patient community.

These organizations are committed to the needs of people and families affected by Sanfilippo Syndrome. The third-party websites, listed below, are provided as a resource only and do not constitute endorsement by Spruce Bio.

The websites below may offer you more information, resources, and guidance. By clicking the links, you will leave the Spruce Bio website and be redirected to a third-party site.

ADVOCACY IMAGES

 

Disclaimer

Spruce Bio is not responsible for, nor does it endorse, the content, terms and conditions of use or privacy practices of external websites.

To learn more about Spruce advocacy or to ask us a question, we invite patient and family caregiver advocates to connect with us at PEL@sprucebio.com.