FAQs

MPS1 is a rare genetic disorder caused by the deficiency of an enzyme called alpha-L-iduronidase. This enzyme is essential for breaking down complex sugars called glycosaminoglycans (GAGs). The deficiency leads to the buildup of GAGs in various tissues and organs, causing a range of symptoms.

Symptoms can vary widely but often include developmental delays, progressive organ damage, skeletal abnormalities, distinctive facial features, joint stiffness, heart problems, vision and hearing loss, and potentially neurological complications..

Currently, there is no cure for MPS1. Treatment focuses on managing symptoms and may involve enzyme replacement therapy, hematopoietic stem cell transplantation, and supportive care to improve quality of life.

Life expectancy varies widely. Particularly Hurler syndrome, can vary significantly based on the severity of the condition and the individual’s response to treatments. Historically, without any treatment, individuals with severe Hurler syndrome might not survive beyond childhood, with life expectancies often ranging into the first decade of life.

However, with early intervention and appropriate treatment, particularly stem cell transplants and Enzyme Replacement Therapy, life expectancy can be extended, although individuals may still face health challenges.

The Kennedy Ladd Foundation endeavors to support families affected by MPS1 by:

• Providing educational resources and support for families navigating MPS1, particularly Hurler Syndrome.
• Offering iPads, to children undergoing bone marrow transplants to ease their journey.
• Fostering a supportive community for families affected by MPS1, encouraging connection and shared experiences.
• Investing in research initiatives aimed at finding a cure and improving treatment options for MPS1.
• Encouraging families to tell their stories and create change.

Statement on Financial Compensation from Co-founders:

At The Kennedy Ladd Foundation, transparency and the responsible use of funds are paramount. We want to assure our donors and supporters that our co-founder, Allie Ladd and Ryan Ladd (The Ladd Family), does not receive any form of financial compensation or remuneration from the foundation.

Our commitment to our cause and the MPS1 community stems from personal dedication and a heartfelt mission to support families affected by MPS1, specifically Hurler Syndrome. Our involvement is entirely voluntary at this time, driven by a desire to make a positive difference and enhance the lives of those impacted by this condition.

Every donation, large or small, is utilized judiciously and directed entirely toward our mission of providing education, support, and hope to families living with MPS1. Your support enables us to provide resources, foster community, and fund critical research without any portion allocated for administrative or personal compensation.

We believe in complete transparency and accountability, and we thank you for entrusting us with your support and generosity. Should you have any further inquiries or require more information, please don’t hesitate to contact us directly.

Thank you for being an integral part of our journey to make a meaningful difference in the lives of those affected by MPS1.