Our Research Strategy
Maximizing Our Investment
As an ultra-rare condition, Multiple Sulfatase Deficiency, or MSD, has historically struggled to attract funding from larger research funding bodies to develop a treatment or cure. The United MSD Foundation aims to ensure this devastating condition receives the research attention and funding it requires and deserves.
To address this lack of funding, the United MSD Foundation has developed a research strategy that:
Aims to maximize the impact of the funds we invest
Identifies research areas that provide the greatest scientific advances toward a cure and the most relevance for our families
Offers research grants for recommended projects following a robust application review process
Our Research Strategy Roadmap
We’re excited to share our newly published Research Strategy Roadmap: a clear, collaborative path for advancing MSD research toward treatments and, ultimately, a cure.
Read the full roadmap in our interactive flipbook here.
Our Research Priorities
Our Research Strategy Roadmap provides a framework for advancing the most promising opportunities in MSD research—from developing potential treatments and preparing for clinical trials to earlier diagnosis, newborn screening, and a deeper understanding of the disease.
We will continue to advance gene therapy while exploring complementary approaches, including drug repurposing, pharmacological chaperones, small-molecule therapies, and ex vivo gene therapy. At the same time, we will invest in the data, biomarkers, clinical tools, and research infrastructure needed to move promising discoveries toward patients.
Because MSD is ultra-rare, collaboration is at the heart of our strategy. By bringing together researchers, clinicians, families, industry, and partners, we can maximize the impact of every research investment and accelerate progress toward treatments and, ultimately, a cure.
From Strategy to Action
The roadmap will guide the Foundation’s research investments over the next 4+ years, helping us identify critical gaps, evaluate emerging opportunities, and prioritize the research with the greatest potential to move MSD closer to treatment.
Our destination is clear: a future where people living with MSD have access to effective treatments and, ultimately, a cure.
Let’s Connect
Connect with MSD families and get valuable information from medical researchers and doctors.