Name: Amber Olsen
Title: Executive director
Organization: United MSD Foundation
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Disease focus: Multiple Sulfatase Deficiency (MSD) is a rare, fatal, lysosomal storage disease that affects the entire body. With MSD, the body does not break down and filter out the natural cellular waste that occurs in everyday cell functions. Children are typically without any symptoms at birth, but depending on their genetic mutation, signs of MSD can begin either soon after children are born or later on in the child’s life. Diagnosis of the disease is very difficult, as there are less than 100 known cases of MSD throughout the world, and there are approximately 20 known cases in the United States. Children are often initially diagnosed with developmental delay and frequent ear infections before receiving a diagnosis of MSD. Children with MSD rarely survive past their tenth birthday, as their entire body shuts down due to a buildup of waste and loss of critical function. MSD is caused by either the absence of or errors within the SUMF1 gene. Over time, cellular waste builds up and is deposited throughout the body in multiple systems. Accumulation of waste products in the brain leads to developmental delay and loss of motor and communication skills. Some children with MSD may talk initially but will eventually lose their verbal skills. Some children with MSD will never develop speech. Other critical body systems are also affected. It may cause blindness, difficulty swallowing and breathing, upper respiratory infections, the need for a feeding tube, curvature or deformation of the spine, joint stiffness, heart conditions, and other problems.
Headquarters: Ocean Springs, Mississippi
How did you become involved in rare disease: My daughter was diagnosed in 2016 with MSD.
Previous career: Entrepreneur. I own a staffing company.
Education: Bachelor of science in business administration from the University of Montana
Organization’s mandate: Cure multiple sulfatase deficiency
Organization’s strategy: We are working like a pharma company. We are finding out all the necessary steps to go into a clinical trial and checking off those items: organizing and educating families, creating a biomarker, developing a biobank, patient registry. This year we plan to fund a prospective natural history study.
Funding strategy: Our board designated our focus on gene therapy, and we are currently only funding work that is on the path to move us to a clinical trial for a gene therapy.
What’s changing at your organization in the next year: We are focused on raising money from major donors that want to be a part of something extraordinary. We are taking next steps toward a clinical trial.
Management philosophy: Keep the mission in front of us. This is about the kids and creating the cure.
Guiding principles for running an effective organization: Keep an eye on the mission always.
Best way to keep your organization relevant: Continuously network and talk with other organizations, support organizations, and talk with researchers and parents.
Why people like working with you: I am tenacious.
Mentor: Other Fighter parents paving the way: Lori Sames, co-founder and CEO of Hannah’s Hope Fund for GAN, who raised $6 million and created a gene therapy clinical trial for giant axonal neuropathy; and Gina Hann, founder and president of Foundation for Batten Hope, who raised funds for a gene therapy clinical trial for the CLN7 form of Batten disease.
On the Job
What inspires you: Thinking about our first family to go to clinical trial be treated.
What makes you hopeful: That this will really work.
Best organization decision: Do what is next. Ask an expert, hire a consultant.
Hardest lesson learned: Have a plan B, and C
Toughest organization decision: Potential commercial partners, what to do.
Biggest missed opportunity: Going with a commercial partner instead of just doing it through a research trial.
Like best about the job: Love our families and kids, doing something so meaningful.
Like least about the job: When a new family gets diagnosed and having to watch their children decline, knowing we could do something.
Pet peeve: Laziness
First choice for a new career: Genetic Counselor
Most influential book: The Four Agreements: A Practical Guide to Personal Freedom by Don Miguel Ruiz
Favorite movie: The Proposal
Favorite music: Meditation music (currently)
Favorite food: Sushi
Guilty pleasure: Netflix
Favorite way to spend free time: Netflix