The National MPS Society is delighted to announce that Sharon King has joined the team as our inaugural Chief Operating Officer (COO). Sharon comes to us with over two decades of experience spanning rare disease nonprofits, industry, and patient advocacy. She is a passionate leader who has dedicated her life to serving patient communities and driving meaningful change in the field of rare diseases.
Terri L. Klein, President and CEO at the National MPS Society, shared, “We are honored to welcome Sharon King as our new Chief Operating Officer. Sharon brings unmatched experience, heartfelt purpose, and a legacy of advocacy that resonates deeply with our mission. Her leadership is shaped by her lived experience and a deep understanding of the challenges faced by families with rare diseases. Her ability to lead with both strategy and compassion will strengthen every facet of our work. I am confident that Sharon will help the Society reach new heights as we continue to serve and support our families. We are thrilled to welcome her and look forward to the strength she will bring to the Society.”
Sharon is a longtime patient advocate and thought leader in rare disease, known for her ability to unite public officials, researchers, biotech representatives, industry leaders, and other advocates in the pursuit of rare disease treatment development and impactful public policies.
Her journey in advocacy began with the founding of Taylor’s Tale, an organization she co-founded in honor of her late daughter, Taylor, to raise awareness and funding for CLN1 disease (a form of Batten Disease) research. Under her leadership, Taylor’s Tale grew from a grassroots initiative into a force in the fight against rare diseases.
Beyond her work with Taylor’s Tale, Sharon has served in key roles that reflect her dedication to improving the lives of rare disease patients and caregivers. As Senior Lead of Advocacy at Aldevron, she worked to educate, connect, and improve understanding across the stakeholder community contributing to groundbreaking advancements.
Sharon is a state-appointed member of the N.C Advisory Council on Rare Diseases. The law that established the Council—the nation’s first—is named for her daughter. She is also a former member of the ASGCT Patient Education committee and serves on the Emily Whitehead Foundation Board of Directors. Her work reflects her deep understanding of the unique challenges faced by rare disease patients and their caregivers, as well as her unwavering commitment to promoting patient-centered, integrated care networks. Her efforts are grounded in personal experience and driven by a vision of improving lives through collaboration, education, and innovation.
As Chief Operating Officer, Sharon King brings a powerful combination of strategic leadership, lived experience, and deep compassion to the National MPS Society. Her expertise will be instrumental in advancing our mission to support individuals and families affected by MPS and ML. With her unwavering commitment to advocacy and innovation, Sharon will help guide our efforts to expand critical services, drive research, and strengthen the communities we serve.
This strategic appointment comes at a pivotal moment as the Society works to close the treatment gap for all families impacted by MPS and ML. With Sharon in this vital role, our team is better positioned to move forward with intention—developing new resources, enhancing care pathways, and ensuring families are never alone in their journey from diagnosis to treatment and beyond. The National MPS Society remains a steadfast partner for the road ahead. Together, we will work toward a brighter future for those we serve.
Sebin, the third of four siblings, has spent nearly her entire life in the hospital, including time in the NICU and ICU. She has now been hospitalized for four years due to the many complications that come with her diagnosis, MPS III, also known as Sanfilippo Syndrome.
“Every day, I fall asleep on an oxygen respirator,” said Hyemin, Sebin’s mother. “The oxygen saturation, increased liver levels, and numerous other symptoms of MPS always make caring for Sebin difficult.”
According ot Hyemin, though, caring for Sebin is no trouble for a family who loves her dearly.
“She lives with a lot of care from her siblings,” Hyemin said. “She is a lovely daughter to mom and dad, a princess to her brothers, and the prettiest princess to her sister.”
Through the ups and downs of a Sanfilippo diagnosis, Sebin remains the heart of her family. “She is always smiling lovingly in the process of struggling, and she is the happiness of my family,” said Hyemin. “Sebin is a princess of my family.”
“I was misdiagnosed at age seven with Spondyloepiphyseal dysplasia after early signs of skeletal differences.. By age eight, I underwent a hip shelf procedure, followed by double hip replacements at 12. At 15, I had a multilevel spinal fusion, and at 38, I had a total knee replacement.
In my early 30s, I began experiencing corneal clouding that significantly impacted my vision and had me looking for more answers. My cornea specialist suggested genetic testing to determine the specific type of corneal dystrophy we had been treating. When the first round of testing came back negative for both Spondyloepiphyseal Dysplasia and Corneal Dystrophy, we laughed and moved on to a second round. During a virtual appointment with a genetic counselor, I was shocked to finally hear the words: Morquio Syndrome, or MPS IV A.
The day after my diagnosis, my research led me to the National MPS Society and the Pathways Program, where I was connected with Evelyn Fisher. It was surprising to learn that even as an adult, I needed to be seen at a children’s hospital, but I was quickly referred to the Children’s Hospital of Philadelphia. Within 30 days of meeting Evie, I started enzyme replacement therapy—ten years after it had become available. Just six months after that, I attended the Orlando MPS Conference, where I met other adults with MPS and found a sense of community I never knew I was missing.
Looking back, I know my parents and doctors did everything right with the information they had—we just didn’t have the diagnosis. I still would have needed the same orthopedic surgeries, but earlier access to enzyme replacement therapy might have made a difference in my disease progression.
To parents who are just beginning this journey: know that your strength and encouragement truly matter. Finding the right physicians and connecting with the MPS community will give you the tools, support, and knowledge to advocate for your child every step of the way. My journey has looked a bit different than most MPS kids, but I still went to college, earned a master’s degree, bought a house, and worked full time up until very recently. A diagnosis doesn’t limit your potential—it empowers you to fight for it.”
Durham, NC — April 15, 2025 — The National MPS Society welcomes the news that Spruce Biosciences is now leading the development of the TA-ERT program (formerly AX 250), bringing renewed hope and momentum to the MPS IIIB (Sanfilippo) community.
Spruce Biosciences has made clear its intention to move swiftly and responsibly, acknowledging the long-standing advocacy and involvement of families, researchers, and clinicians. The company recognizes the challenges and uncertainty caused by the previous pause in development and is committed to rebuilding trust through open, consistent engagement.
“We are encouraged and deeply grateful to see Spruce Biosciences advancing the TA-ERT program for individuals living with MPS IIIB, also known as Sanfilippo Syndrome,” said National MPS Society President & CEO Terri Klein. “This marks an important step forward in addressing an urgent, unmet need for this patient community, and we commend the renewed commitment to those impacted by MPS IIIB. The National MPS Society remains hopeful and supportive as this promising therapy moves toward clinical development and, ultimately, to the families who are waiting.”
Manufacturing of TA-ERT has resumed to ensure the availability of products, and plans have been made to pursue accelerated approval in the U.S., initiate a registrational clinical study, and expand global early access opportunities by early 2026.
Community partnership is a central pillar of Spruce Bioscience’s approach. A dedicated Patient Engagement function has been established, and additional opportunities for communication, feedback, and collaboration will be available to the patient community.
The National MPS Society is encouraged by Spruce Biosciences’ renewed dedication to this program, and we look forward to continuing to work together on behalf of families living with MPS III.
To read Spruce Bioscience’s full statement, please click here.
In 2020, in the quiet hum of a hospital NICU, Lauryn and her husband began a journey no parent ever anticipates. Their newborn daughter, Blakely Mae, had just been diagnosed with Mucolipidosis (ML) Type II, a rare and devastating genetic disorder. In that moment, their world shifted—ushering them into the role of “rare parents.”
“In the midst of overwhelming emotions, uncertainty, and grief, we made a choice to live each day with love,” Lauryn recalled. “And to pack a lifetime of love into the short time we had with Blakely.”
And that’s exactly what they did. Blakely’s life, though heartbreakingly brief, was radiant. She lived just two and a half years, but every moment was saturated with affection, tenderness, and joy.
The loss of Blakely was shattering. “To say we were devastated is an understatement,” Lauryn shared. “We would have moved mountains to heal her. But now, we’re trying to find purpose in the pain.”
Since Blakely’s passing, Lauryn and her family have been committed to honoring Blakely’s memory by raising awareness for ML and advocating for other children facing rare diseases. “Every day, we honor her name, her spirit, and the other rare children who still need a cure.”
Blakely Mae may have only been here for a short time, but her impact endures—a testament to the powerful love of her family and the joy she brought to all who knew her.
“We miss you more than words can express, Blakely Mae,” said Lauryn. “You are forever in our hearts.”
The National MPS Society expresses deep appreciation and concern following the announcement that Dr. Peter Marks will resign from his role as Director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER). In a letter to the FDA Dr. Marks stated that his departure will be effective April 5, 2025.
This leadership change is a significant loss for the rare disease and MPS communities, and it comes as a significant blow to all who rely on stable, expert-driven regulatory oversight. Dr. Marks has been an invaluable champion for patients with Mucopolysaccharidoses (MPS), and his resignation leaves a profound void in the effort to develop and approve life-saving therapies for rare diseases.
During his nine-year tenure leading CBER, Dr. Marks oversaw the approval of 22 novel gene and cell therapies – groundbreaking treatments that have brought hope to families facing conditions once thought untreatable.
Dr. Marks’ personal commitment to the MPS community was evident in his direct engagement with patients and families. He delivered a keynote address at the National MPS Society’s 37th Annual Family Conference in 2023 in a session aptly titled “Addressing the Unmet Needs of the Rare Disease Community.” At this event, Dr. Marks spoke candidly and empathetically about the challenges of developing therapies for ultra-rare diseases. He exhibited a deep understanding of and sympathy towards MPS families in his comments during the panel session.
By taking the time to engage at the conference, Dr. Marks demonstrated extraordinary commitment to those living with MPS, reinforcing the principle that patients and caregivers must be partners in the drug development process. He recognized that the voices of patients and their families are central to regulatory decisions, and worked relentlessly to ensure those voices informed and shaped the path of drug development. His leadership fostered innovative gene therapy programs while instilling a culture of compassion in the regulatory process, directly benefiting the MPS community and beyond.
Moving Forward – Protecting Progress and Calling for Continued Leadership:
Dr. Marks’ resignation comes at a pivotal moment for rare disease research and regulation. The National MPS Society reaffirms our commitment to protect the progress we have found so hard to achieve in rare disease drug development. We will continue to work tirelessly with policymakers, regulators, and industry partners to ensure that the momentum in developing therapies for MPS and ML remains uninterrupted.
Our community cannot afford delays or disruptions. We urge the FDA and the Administration to maintain strong, experienced leadership at the Agency to support the needs of patients with rare diseases, and we stand ready to collaborate with FDA leadership to sustain the uninterrupted momentum in rare disease therapy development so that the innovative treatments on the horizon become realities for the families who need them the most.
About the National MPS Society
Founded in 1974, the National MPS Society is a 501(c)3 non-profit organization dedicated to supporting individuals and families affected by Mucopolysaccharidoses and Mucolipidoses. The Society’s mission is to advance research for cures, provide advocacy and support, and promote awareness of these devastating, progressive diseases.
Contact: Terri Klein, President and CEO
terri@mpssociety.org
“In 2018, our family welcomed our youngest daughter, Raelin, into the world. From the moment she was born, we were completely in love. She lit up our lives with her spunky personality andbeautiful red hair. Despite some early developmental concerns, Raelin seemed to be progressing well as an infant. However, as she grew into toddlerhood, we began noticing increasing developmental delays. We also discovered that she was losing her hearing.
What initially seemed like a slightly delayed speech soon began to regress rapidly. Doctors and specialists were unable to provide a clear explanation for her symptoms. Eventually, she was diagnosed with autism, but to us, it felt like an incomplete answer. She underwent a chromosomal microarray genetic test, which came back normal. As newcomers to the world of genetic testing, we didn’t realize that this particular test wouldn’t screen for MPS III.
Finally, a neurologist at Phoenix Children’s Hospital ordered a full genetic sequencing test. At age six, we received the devastating diagnosis: Raelin had MPS III, also known as Sanfilippo Syndrome.
Through this journey, we have learned to cherish the invaluable gifts of hope and time. Hope for a cure. Hope for a treatment. Time together as a family. Time with Raelin. While this disease is fatal, and we watch as she loses a little more of her abilities each day, we are deeply grateful for her. She is a precious gift, bringing immeasurable joy to our family. Everyday with her is a blessing.
We are truly grateful for the support we have received from MPS Society and Cure Sanfilippo, whose dedication to research, advocacy, and community is truly impacting our family. We are also truly grateful for Raelin’s medical team, compassionate ABA therapists, caring school team, church, and amazing friends and family. Through this journey, we have also met other truly incredible MPS Families. It is community that helps give us the courage to face each day and reminds us that we are not alone in this journey.”
Matthew Wain (MPS I)
The Wain family’s journey with MPS began in 1987 when their firstborn, Matthew, was diagnosed with MPS I. Doctors immediately asked if there were other children, and at just three months old, Matthew’s younger sister, Stacy, was also diagnosed with MPS I. At the time, bone marrow transplants were an emerging treatment. Due to complications from prematurity and weak lungs, Matthew was not a candidate, however Stacy was deemed an ideal recipient.
The Wain family traveled to Iowa, where Stacy became the first patient to receive an unrelated bone marrow transplant at the University of Iowa. Amidst their hope for the future, in 1990 the family also experienced heartbreak: Matthew passed away.
Stacy Wain (MPS I)
Despite the challenges she faced and the loss of her brother, Stacy became a trailblazer for individuals with Hurler syndrome. She graduated from high school, attended classes at a local community college, and embraced independence, eventually living in her own apartment with her beloved cat.
In 2014, Stacy underwent surgery for her gallbladder and spleen. Sadly, six days later, she passed away due to complications from the surgery.
Stacy’s mother, Nancy Wain, remembers her as spunky and spirited, and said that her laugh could light up a room.
“The MPS Society was a blessing to our family with the support it offered,” said Mrs. Wain. “Meeting new friends at conferences made us feel like we weren’t alone, and for that we are grateful.”
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“MPS II, also known as Hunter Syndrome, has been a part of my life for as long as I can remember. I grew up watching my little brothers battle this devastating disease, witnessing their joy turn into struggle as they slowly lost the abilities most of us take for granted. First, their words faded. Then, the ability to eat, to walk, to breathe. I held their hands as they slipped away—Nathaniel at just 11 years old, and three years later, Nyaire at 14. Their lives were stolen by this cruel disease, and the grief of losing them still weighs heavy on my heart.
But my story didn’t end there.
Years later, I became a mother. My son PJ was born on April 1, 2017. A vibrant, curious, brilliant baby who hit every milestone—some even ahead of schedule. I called him my genius baby. But shortly after his first birthday, things changed. Frequent ear infections, severe sleep apnea, hospitalizations for breathing issues. Doctors reassured me, offering tips to fight daycare germs. Then, I became pregnant with Pharaoh. Almost immediately, he too struggled to breathe.
One month before PJ’s second birthday, our world shattered. My thriving, energetic little boy suddenly lost all his skills. Walking exhausted him. He stopped sleeping. And when I looked at his face, I saw something eerily familiar—his features were changing, just like my brothers’. My heart pounded as I picked up the phone to call a geneticist.
The moment the doctor and genetic counselor walked into the room, I knew.
“I’m almost certain he has Hunter Syndrome,” they said, confirming my worst fear. “We need to test Pharaoh too.”
And just like that, my sons’ futures—their hopes, their dreams—were stolen before my eyes.
Grief introduced itself to me again. But this time, it was different. I was grieving my beautiful boys while they were still alive, knowing MPS II would take everything from them.
Shortly after, PJ was hospitalized with rhinovirus and placed on oxygen. What was supposed to be a short stay turned into two months; during that time, our incredible genetic counselor set us up with every specialist imaginable. I juggled appointments for Pharaoh, then rushed back to PJ’s hospital room, repeating this cycle day after day. Cincinnati Children’s Hospital became our home.
Then, a glimmer of hope.
Our genetic counselor found a clinical trial at UPMC Children’s Hospital of Pittsburgh. It wasn’t a cure, but it was a chance—something my brothers never had. For months, we traveled between Cincinnati and Pittsburgh, just me and my boys. Eventually, both PJ and Pharaoh were accepted into a clinical trial. I packed up our lives and moved to Pittsburgh, determined to give them a fighting chance.
In August 2022, life threw me another curveball—I was pregnant again. My third son was born in May 2023 and was tested for Hunter Syndrome at birth. The result? Positive. My heart shattered once more. But this time, we had a plan. Unlike his brothers, he started treatment at just three months old.
All three of my sons are part of a clinical trial, and while they are doing well, this is not a cure. The treatment is not FDA-approved, meaning many families don’t even have access to this small chance at hope. The trial may slow some of the disease’s effects, but it cannot undo the damage already done. My oldest boys, now seven and six, are nonverbal, not potty trained, and struggle with behaviors rooted in frustration and pain they cannot communicate.
We need a cure.
I planned two funerals for [my brothers], and the thought of losing my sons the same way breaks me. No parent should have to live in constant fear of a ticking clock, watching time slip away, knowing they are powerless to stop it.
We need newborn screening. We need FDA approval for existing treatments so more families can access them. We need funding for research. We need hope.
MPS is stealing our children. Their futures should not be just a dream. It should be a reality!”
