We are deeply grateful to the individuals, organizations, and supporters who have contributed their time, talent, and resources to the mission of SMA-PME Research.
3rd ACDase Deficiency Global Forum
We extend our deepest gratitude to the distinguished speakers and participants who contributed their time, expertise, and dedication to the Third Acid Ceramidase Deficiency Global Forum held on April 28, 2026. Their commitment to advancing research and fostering global collaboration brings us closer to life-saving treatments for the children we fight for.
- Dr. Reem Eissa — The Fight Farber Foundation
- Dr. Nicole Chemaly — Clinician presentation on SMA-PME
- Dr. Fengfeng Bei et al — AAV.CPP.16-based ASAH1 Gene Supplementation
- Dr. Diego Medina et al — High Content Phenotype Screening for Drug Repurposing in Farber Disease
- Dr. Mathias Schmidt — Strategies of Overcoming the Blood Brain Barrier
- Dr. Michelle Allen-Sharpley — Lentivirus Mediated Gene Therapy for the Treatment of Acid Ceramidase Deficiencies
- All Forum Participants — Families, researchers, and clinicians from around the world
Recent Recognition
We thank Ms. Lisa Eliot with Pampered Chef, LLC for her generous donation of valuable time, energy, and funds to support our charity during an August 2025 and all follow-on FUNdraisers.
2nd ACDase Deficiency Global Forum — November 13, 2023
We are deeply grateful to the distinguished speakers and all participants who contributed their time and expertise to the Second Acid Ceramidase Deficiency Global Forum.
- Ms. Mary Irwin — Overview, SMA-PME Research
- Dr. Ed Schuchman — Overview: Acid Ceramidase and ERT Status Update
- Dr. Jeffrey Medin — Cell Therapies for ACDase Deficiencies in Mouse Models
- Dr. Silvestre Cunait — Natural History of SMA-PME Based on 9 French and American Patients
- Dr. Nils Confer — Birth Prevalence of SMA-PME and Farber Disease
- All Forum Participants — Families, researchers, and clinicians from around the world
1st ACDase Deficiency Global Forum — June 26, 2023
Heartfelt thanks to the international research scientists and leaders — including American, Canadian, and researchers from around the world, as well as the French charity ASAP for Children — who participated in our inaugural global forum on acid ceramidase deficiency.
Birthday Benefit
We extend our gratitude to Mark Schratz of Chandler, Arizona, who requested donations to SMA-PME Research instead of birthday gifts in January 2023.
Benefit Concert Contributors
Special thanks to the following organizations for their support of our March 27, 2022 Benefit Concert:
- Trinity United Methodist Church
- Coastal Carolina University Department of Music
- Grand Strand American Guild of Organists
- Allspice Studios (Myrtle Beach, SC) for graphic design services
Continuous Support
We are grateful to Dr. Jeffrey Medin from Medical College of Wisconsin for his consultation and website review assistance.
We extend deep appreciation to all parents of children with SMA-PME and Farber disease who have shared their children's stories on our website.
We thank our anonymous donors who made personal, private contributions — entirely separate from public charity funds — to help Adeline's family purchase essential mobility equipment, including a transport chair and electric wheelchair. Our public charity funds support all children in our community, not any single family.