Maria Kefalas, Dean Suhr and Teryn Suhr to receive the 2025 Patient Advocate Leader (PAL) Award

After reviewing numerous nominations, and considering many amazing individuals, the WORLDSymposium 2025 Awards Committee selected Maria, Dean and Teryn as the recipients of the 2025 PAL Award.

Maria Kefalas studied economics at Wellesley College and earned her MA and PhD in sociology from the University of Chicago.  With her late husband Pat Carr, Dr. Kefalas is the co-founder of the Calliope Joy Foundation which provided funding to help launch the Leukodystrophy Center of Excellence at the world-renowned Children’s Hospital of Philadelphia. Over a decade, the Calliope Joy Foundation raised almost $1 million, which was used in part to send 20 children to Italy where they received gene therapy for metachromatic leukodystrophy (MLD). She worked with families from Brazil, Australia, and New Zealand to get children treated through the clinical trials, and she’s been on a mission to get the treatment approved. She is a nationally recognized parent advocate for gene therapy who has spoken at the NIH, NORD, and the FDA. 

As executive director of Cure MLD, she helped organize a listening session with the FDA in 2019 and later a Patient Focused Drug meeting with the agency. Although it was too late for her daughter Calliope (Cal), Dr. Kefalas championed the clinical trials and the therapy, and the FDA recently approved the first gene therapy to treat MLD.

Dean Suhr, BS, is the MLD Foundation President & co-Founder and Teryn Suhr, RN, is the MLD Foundation Executive Director and co-Founder. Their advocacy work over the past 20+ years has included involvement in over a dozen MLD research projects, including substrate reduction, gene, and enzyme replacement therapies. They facilitated the transfer of the MLD mouse model to the US to support numerous studies. Teryn and Dean continue to be actively engaged in efforts to implement MLD newborn screening both in the US and beyond. On Capitol Hill they are working to advance rare disease biochemical, public health, access and reimbursement, privacy, genomics, newborn screening, and other rare disease policies & regulations.

Dean and Teryn are parents to three girls, two of whom were diagnosed with MLD in 1995 after a six-year diagnostic odyssey. They lost their youngest daughter, Darcee, at ten years old due to complications after a BMT and are actively caring for their 44-year-old daughter, 30 years postdiagnosis, who continues to regress day by day.

Darcee’s death was the catalyst to plunge them into the work they have done for the last 2+ decades for Metachromatic Leukodystrophy. Upon realizing MLD was also a lysosomal disease and the biochemistry was more closely related to the LSDs, they turned to learning more about the LSDs and exploring possible connections in the Lysosomal space. The Suhrs have been attending, educating, and networking at the WORLDSymposium™for the last 20 years. Dean and Teryn have a long history of supporting and working with their LSD peers through the Symposium’s COPA and CPAG groups and collaborating with LSD groups like the MPS society and MSD community on projects.

The Suhrs are published authors on numerous scientific publications on topics such as Leukodystrophy Standards of Care, MLD NBS assay mechanism, MLD genotype-phenotype correlation & prediction, and have published numerous policy & editorial statements.

Teryn is a retired pediatric and newborn intensive care Registered Nurse. Dean is a retired hightech engineer, business development, marketing, sales, and operations executive applying decades of those skills to rare diseases. A more detailed bio is available at the MLD Foundation website.

In recognition of their tireless efforts, Dr. Kefalas and the Suhr’s will receive the Patient Advocate Leader Award on Wednesday, February 5, 2025, at 08:00 PST, at WORLDSymposium 2025.

About the WORLDSymposium Patient Advocate Leader (PAL) Award

Each year, WORLDSymposium recognizes individuals for patient advocacy leadership in the field of lysosomal disease.

An announcement of the award recipient is included in the February lysosomes issue of Molecular Genetics and Metabolism. (This award is given on a yearly basis, but can be omitted in any given year at the discretion of the Planning Committee.) Nominations are solicited annually from members of the lysosomal disease community, planning committee members and attendees of WORLDSymposium.

The 2025 Patient Advocate Leader Award will be presented at 08:00 PST on Wednesday, February 5, 2025, at the 21st annual WORLDSymposium in San Diego, California.

Past Award Recipients:

2016 Barbara Wedehase, National MPS Society
2017 Christine Lavery, UK Society for Mucopolysaccharide Diseases
2018 Jack Johnson, Fabry Support & Information Group
2019 Mark Dant, EveryLife Foundation for Rare Diseases, National MPS Society
2020 Cara O’Neill, MD, Cure Sanfilippo Foundation
2021 Terri L. Klein, NPGC, National MPS Society, USA
2022 Sue Kahn, National Tay-Sachs & Allied Diseases Association (NTSAD)
2023 Christine Waggoner, Cure GM1 Foundation
2024 Alan Finglas, MSD Action Foundation & SavingDylan.com