Team Sanfilippo Trials

For parents caring for a child with a Sanfilippo diagnosis, understanding the path to new treatments is crucial. Team Sanfilippo Foundation aims to accelerate this journey and share important updates on how potential therapies are moving forward.

The Path to Treatment: Regulatory Designations

When a new treatment for Sanfilippo Syndrome is developed, it needs special recognition from health authorities to help speed up its review and approval. We’re excited to share that a promising compound, SLS-005, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

What does this mean for your child? This special status is granted to treatments for rare diseases, like Sanfilippo Syndrome. It signals that these agencies recognize the urgent need for a therapy and are committed to supporting its development. It helps streamline the process, getting potential treatments to children faster.

Seelos Therapeutics has received the US Orphan Drug Designation for SLS-005 (Trehalose) in Amyotrophic Lateral Sclerosis. The promising substance, Trehalose, is generating interest across the field of neurodegenerative diseases.

Advancing to Clinical Trials: Testing New Possibilities

The next critical step for SLS-005 is a combined Phase IIb/III clinical trial. This extensive study is being developed to evaluate the treatment’s safety carefully, how well patients tolerate it, and most importantly, how effective it is for individuals with Sanfilippo Syndrome types A, B, C, and D. This trial isn’t just about the medication; it’s about real-world impact. Researchers will be closely monitoring.

Functional Outcomes: Clinical trials are needed to comprehend how the treatment affects daily activities and abilities.
Biomarkers: Specific indicators in the body that show how the disease is responding or progressing to the treatment.
Neuro-cognitive Assessments: How the treatment influences thinking, learning, and memory.
Quality of Life Measurements: Understanding the overall well-being and happiness of the children and their families.

IV Trehalose

What Is Trehalose (SLS-005)?

Trehalose is a naturally occurring disaccharide sugar that has shown potential in stabilizing proteins and activating autophagy, a cellular process that helps clear damaged components. In preclinical studies, SLS-005 demonstrated the ability to cross the blood-brain barrier, reduce inflammation, decrease nerve cell degeneration, and extend lifespan in mouse models of Sanfilippo syndrome. ALS News Today+1Neurology live+1Sanfilippo Syndrome News.

Thanks to your support, Team Sanfilippo Foundation is actively funding and running crucial clinical trials designed to find a cure for Sanfilippo Syndrome. To ensure these trials continue and succeed, we are asking the participation of more families.

If you are a family living with Sanfilippo Syndrome and are interested in learning how to enroll in our current trials, please contact Kathleen Buckley at 518-879-6571.

You can directly contribute to the scientific breakthroughs that will change the lives of Sanfilippo children. Together, we can give them a chance at a brighter future.

Expanded Access: A Glimpse of Hope

We are pleased to inform you that two patients are currently receiving treatment with SLS-005 under the Expanded Access program in the United States and Switzerland.

At Team Sanfilippo Foundation, every step forward in clinical development brings us closer to the treatments our children urgently need. We are committed to keeping you informed as these promising therapies progress.