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Patient Partners: Harnessing synergies to pursue ASOs as part of the AHC research portfolio

Monday January 29, 2024 12:30 PM US EST





Mary will share her son Henry's case and describe her efforts to develop an n=1 ASO for

him. Nina will contextualize ASO development within the wider scope of the AHC research

program, and discuss strategy for patient foundation investment across a portfolio of

therapeutics-focused studies. Mary and Nina will highlight the advantages of advancing both

N=1 and AHC community studies jointly, collaboratively, and in parallel, as partners with

researchers. Dr. Cat Lutz, Vice President of the Rare Disease Translational Center at the

Jackson Laboratory, will share details of her team's efforts to establish proof of concept for

AHC therapeutics in mouse models of AHC.




Nina Frost

Mom of Annabel (and Clara)

President, Hope for Annabel


Nina leads the research program for Alternating Hemiplegia of Childhood (AHC). Nina has

worked to centralize, design, and oversee a portfolio of therapy-centric research efforts on

behalf of the AHC patient community.


Nina and Simon Frost founded Hope for Annabel in 2018 to help catalyze research for

therapies for AHC, and to serve as a research hub for the community. Hope for Annabel

works closely with other national AHC organizations, and maintains close ties to researchers

in the field.


Nina has led Hope for Annabel’s fundraising efforts since 2018. Nina also led Hope for

Annabel’s successful grant application for the $2 million Chan Zuckerberg Initiative award in

2022.


Nina’s prior professional experience was in management consulting. Nina and Simon met at

Cambridge University, where they were graduate students together. They now have two daughters,

Clara (10) and Annabel (7). Nina now works full time on AHC.


Dr. Cat Lutz, Ph.D

As the Vice President of the Rare Disease Translational Center (RDTC) at The Jackson

Laboratory (JAX), Dr Lutz works with patient-based foundations, researchers, and industry

partners to roadmap therapeutic strategies for rare diseases. Her work involves the engineering of

preclinical mouse models that represent patient-based mutations and the subsequent phenotyping

of the mice to explore clinically relevant, translatable phenotypes.


Mary Saladino

Henry’s mom

President, For Henry AHC

VP Research, Cure AHC


Mary is a member of the research team in the AHC community and leads research for the

development of an ASO for her son Henry.


Mary and her husband Anthony founded For Henry AHC in 2022 with the goal of raising

funds to advance a treatment for Henry with a current focus on ASO development. For

Henry AHC has raised $1.5M towards this effort to date.


Mary works closely with our national AHC foundations to build relationships with leading

experts and industry partners and to support research projects. Mary shares our research

team’s commitment to supporting our AHC community at large.


Before holding her most esteemed title of being Henry’s mom, Mary was a high school

English teacher and assistant principal. Mary holds a Master’s degree in education and

leadership from Stanford University and is a former Fulbrighter. Mary has embraced the

pivot from championing equity in education to pursuing therapies for AHC and works full time

on this endeavor and in caring for Henry, who is now 4.




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