Catie Becker, NP | Pediatric Nurse Practitioner
Catherine (Catie) Becker is a Pediatric Nurse Practitioner at Massachusetts General Hospital (MGH). Catie received her master’s degree in Pediatric Acute/Chronic Advanced Practice Nursing from the University of Pennsylvania. She has spent the last 15 years working in pediatric neurology at MGH as. Catie joined the MGH Leukodystrophy clinic in 2011. Since 2011 she has been fortunate to work with a multidisciplinary team of care providers treating both children and adults with leukodystrophy, while participating in multiple ongoing clinical trials. Catie looks forward to the Hunter’s Hope meeting every year where she leaves feeling even more inspired and determined to push care forward for every individual living with a leukodystrophy.
Annamarie Dillon | Executive Director Global Patient Advocacy, Orchard Therapeutics
Annamarie Dillon is a passionate advocate for rare disease communities, with over 17 years of experience in industry-based patient advocacy. She is dedicated to incorporating the patient perspective throughout the drug development process improving understanding of the patient journey and informing key decisions such as clinical trial design and data generation for regulatory and HTA submissions.
Annamarie actively builds mutually beneficial partnerships with patient organizations around shared priorities, including disease awareness, newborn screening, community capacity building and networking.
Christine Duncan, MD | Director, Gene Clinical Research and Clinical Development, Boston Children’s Hospital
Dr. Christine Duncan focuses on the cellular therapy of children with rare, nonmalignant disorders and the and the long-term effects of pediatric hematopoietic cell transplant (HCT) and gene therapy. Dr. Duncan has been in an
attending and clinical researcher in this space since 2007.
During that time she has served in multiple leadership capacities at our center and in international consortia. The current focus of her work is the cellular therapy of children with rare neurologic and2 metabolic diseases with an emphasis on patients diagnosed with adrenoleukodystrophy. Dr. Duncan has led multiple international studies of gene therapy for boys with the cerebral form of this rare, X-linked disease.
Michael Gelb, PhD | Boris and Barbara L. Weinstein Endowed Chair, University of Washington
Dr. Michael H. Gelb is the Boris and Barbara L. Weinstein Endowed Chair in Chemistry in the Department of Chemistry and Department of Biochemistry at the University of Washington. He holds a Ph.D. from Yale University, and was an American Cancer Society Postdoctoral Fellow at Brandeis University with Professor Robert H. Abeles. In 1985, he joined the faculty of chemistry at the University of Washington. The Gelb research laboratory combines chemical and molecular and cellular biochemistry techniques to study enzymatic processes of medical importance.
Major achievements include: 1) Discovery of protein prenylation; 2) The development of Isotope-Coded Affinity Tags (ICAT) for quantitative proteomics; 3) The development of assays for newborn screening of metabolic diseases; 4) Development of drugs to treat tropical parasite diseases; 5) Discovery of phospholipases A 2 and understanding their mode of action. In the area of newborn screening, assays for approximately half of the diseases added to the Recommended Newborn Screening Panel (RUSP) over
the past decade have been developed in the Gelb laboratory.
He has received a number of awards including the Repligen Award and the Pfizer Award in Biological Chemistry (American Chemical Society), the ICI Pharmaceuticals Award for Excellence in Chemistry (AstraZeneca, Inc.), the Gustavus John Esselen Award (Harvard University), Merit Award (National Institutes of Health), Medicines for Malaria Venture Project of the Year (MMV, Geneva), Guthrie Award (International Society of Neonatal Screening), and the University of Washington Faculty Lecture Award. He is a Fellow of the American Association for the Advancement of Sciences and a Fellow of the Alfred P. Sloan Foundation.
Joanne Kurtzberg, MD | Jerome Harris Distinguished Professor of Pediatrics
Dr. Joanne Kurtzberg is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood and birthing tissues in the emerging fields of cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant and Cellular Therapy Program, Director of the Carolinas Cord Blood Bank, and Co-Director of the Stem Cell Transplant Laboratory at Duke University.
The Carolinas Cord Blood Bank is an FDA licensed public cord blood bank distributing unrelated cord blood units for donors for hematopoietic stem cell transplantation (HSCT) through the CW Bill Young Cell Transplantation Program. The Robertson GMP Cell Manufacturing Laboratory supports manufacturing of RETHYMIC (BLA, Enzyvant, 2021), allogeneic cord tissue derived and bone marrow derived mesenchymal stromal cells (MSCs), and DUOC, a microglial/macrophage cell derived from cord blood.
Dr. Kurtzberg’s research in MC3 focuses on translational studies from bench to bedside, seeking to develop transformative clinical therapies using cells, tissues, molecules, genes, and biomaterials to treat diseases and injuries that currently lack effective treatments. Recent areas of investigation in MC3 include clinical trials investigating the safety and efficacy of autologous and allogeneic cord blood in children with neonatal brain injury – hypoxic ischemic encephalopathy (HIE), cerebral palsy (CP), and autism. Clinical trials testing allogeneic cord blood are also being conducted in adults with acute ischemic stroke. Clinical trials optimizing manufacturing and testing the safety and efficacy of cord tissue MSCs in children with autism, CP and HIE and adults with COVID-lung disease are underway. DUOC, given intrathecally, is under study in children with leukodystrophies and adults with primary progressive multiple sclerosis.
In the past, Dr. Kurtzberg has developed novel chemotherapeutic drugs for acute leukemias, assays enumerating ALDH bright cells to predict cord blood unit potency, methods of cord blood expansion, potency assays for targeted cell and tissue based therapies. Dr. Kurtzberg currently holds several INDs for investigational clinical trials from the FDA. She has also trained numerous medical students, residents, clinical and post-doctoral fellows over the course of her career.
Hannea Milliman, MS | Social Work and Dance
Hannea Milliman is a recent graduate of Nazareth University, where she earned her Master’s degree in Social Work after completing dual undergraduate degrees in Dance and Social Work. Her academic and professional path reflects her lifelong passion for movement and her deep commitment to supporting and empowering others.
Hannea’s story is one of resilience and perseverance. When Hannea was seven years old, she suddenly experienced paralysis in her right arm due to a rare condition known as Parsonage-Turner Syndrome with Right Brachial Plexus Neuritis. Later in her medical journey, it was determined that this condition was caused by Acute Flaccid Myelitis (AFM). Her world changed as she had to adjust to using only her left arm in everyday life.
Despite the physical limitations, Hannea remained determined to continue dancing. She redefined what movement meant to her and deepened her connection to the art form. Dance became her outlet, her strength, and her way to heal. Throughout her adolescence and college years, she continued to perform and choreograph, exploring new ways of moving and discovering what it truly means to be a dancer with a disability.
In addition to her dedication to dance, Hannea is equally passionate about social work. The care and support she received throughout her medical journey, especially from Philadelphia Shriners Hospital for Children, inspired her to follow a path where she could offer others the same sense of support and empowerment.
As a social worker and lifelong dancer, Hannea is dedicated to combining her passion for movement with her drive to make a difference. She believes that dance is for everyone, regardless of ability, and that everybody and every story deserves to be seen and celebrated. She’s passionate about creating spaces where people feel empowered, connected, and supported. As she grows in both fields, she looks forward to helping others heal through dance and human connection.
Robert Thompson Stone, MD | Pediatric Neurologist
Dr. Robert Thompson Stone is a pediatric neurologist with special expertise in caring for children with immune conditions affecting the neurologic system and those with inherited disorders of white matter (leukodystrophy). He currently serves as Director of the Pediatric Neuroimmunology and Multiple Sclerosis Program and Director of the UR Leukodystrophy Care Center.
Amy White, MS, CGC | Genetic Counselor
Amy White is a laboratory genetic counselor at Mayo Clinic Biochemical Genetics Laboratory, where she specializes in diagnostic testing of inborn errors of metabolism, communication with physicians and other clinicians, and scientific education and research activities. Ms. White completed her undergraduate degree at Lawrence University and obtained a master’s degree in medical Genetics-Genetic Counseling from the University of Wisconsin-Madison. She is board-certified by the American Board of Genetic Counseling and a licensed genetic counselor in Minnesota and Wisconsin.
Ms. White has an academic rank as an Assistant Professor of Laboratory Medicine and Pathology in Mayo Clinic College of Medicine and Science. Prior to this, she was a clinical genetic counselor in the Metabolic Genetics Clinic at Children’s Wisconsin for over 12 years. She has been actively involved in newborn screening programs in both Wisconsin and Minnesota for a combined 20 years. Amy’s most recent work has focused on the collection and analysis of data on biochemical analyses in individuals with Krabbe disease.
Sensory Sisters
Kristen Malfara, Deborah Hecker, Jennifer Fitzgerald
Kristen and Morgan Malfara are from Ocala, Florida. Morgan is now 27, and has Aicardi-Goutieres Syndrome (AGS). Kristen is the founder of The M.O.R.G.A.N. Project and spent 22+ years serving the disability community with unique and innovative multi-sensory programs and providing small grants to families.
In 2023 they partnered with Hunters Hope when they established The Morgan’s Legacy Gift Endowment Fund to provide perpetual financial support for the Morgan’s Legacy Gift Program, which supports families with a gift of up to $1,000 for something that will enhance their quality of life, and provide the tools to help a parent help their child. Kristen has been a member of the LCN Steering Committee since its inception, and supports the annual Family Symposium by creating a Sensory Room for the families to enjoy. This year she is overjoyed to be bringing Morgan with her!
Debbie Hecker is from New London, Connecticut and she and Kristen have been best friends for over 50 years! Jen Fitzgerald is from Melbourne, Florida and in addition to her friendship with Kristen and role on the board of The M.O.R.G.A.N. Project, she was Morgan’s longtime Occupational Therapist. Last year Deb & Jen joined Kristen at Symposium for the first time and the Sensory Sisters were born!
This year, in addition to the Sensory Room itself, the Sensory Sisters will be sharing their expertise with a hands-on presentation and demonstration of OT techniques to support the multi-sensory approach. Additionally, they will be poolside several times throughout the week for OT in the pool. There will also be a sign-up sheet for anyone that would like to do a one-on-one with Jen for creative OT ideas for working with your own child or to discuss ideas with Kristen for creating a sensory space in your own home for your child.
Please join us in the Sensory Room for some therapeutic fun!
Kristen Malfara - Sensory Sister
Kristen Malfara is the proud mother of Morgan, who in 1997 was born with a rare neuromuscular disorder classified as a Leukodystrophy. She is also the Founder of The M.O.R.G.A.N. Project which was established in 2001. Morgan is the inspiration and namesake of this organization. In 2023, Kristen made the bittersweet decision to dissolve the organization. The M.O.R.G.A.N. Project established an endowment with Hunter’s Hope so that Morgan’s legacy can live on and continue to provide hope and support to the rare disease and disabled community nationwide. Morgan is now 26 years old. In addition to Morgan, Kristen is the proud parent of two other sons, Jordan and B.J.
While she still refers to herself as a “stay at home mom,” Kristen remains a leader in the local disabilities community and beyond. The M.O.R.G.A.N. Project was a labor of love for over two decades. The organization helped many families of children living with diagnoses of so-called orphan diseases who need help adapting to life in a world not designed for them. Kristen believes “that our choices, more than our abilities, show who we really are”, and that every family she helped is just one more way that she gets to honor Morgan. Kristen and The M.O.R.G.A.N. Project have been recognized and featured in local and national media outlets, and have been awarded numerous honors over the years.
One of Kristen’s proudest accomplishments has been joining the Hunter’s Hope Leukodystrophy Care Network Steering Committee. She believes that this is her opportunity to come full circle and give back to those that were there for her when her own son was diagnosed many years ago.
