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Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting

Monday, March 4, 2024 12:30 pm US EST

From an initial email request or conversation at a meeting… to (for some) the possibility of additional assessments… to (for a few) the potential or actual delivery of an n-of-1 treatment to an individual, researchers and clinicians are in contact with patients, families, and caretakers throughout this process. These contact points are crucial for evolving treatment development, but also present challenges on each side. For this webinar, we invite you to join a panel discussion with Marlen Lauffer, Margaret Meserve, and Lynn Bush who will be sharing their experiences in respect to navigating family conversations in n-of-1 settings. If you would like the panel to address a specific question or topic, or if you have an experience you would like to briefly share if time permits, please respond within this form.

Marlen Lauffer, MD is a senior researcher at the Dutch Center for RNA Therapeutics (DCRT). She has a background in biochemistry, human molecular genetics, and medicine, and has been studying rare diseases in and outside of the wet lab for the past decade. Marlen is especially interested in rare neurological disorders manifesting in childhood, like neurodevelopmental disorders and childhood dementias. At the DCRT, Marlen is responsible for patient evaluation and selecting amenable genetic variants for N=1 treatments as well as overseeing the development and testing of antisense oligonucleotides in the wet lab. She is co-leading the N1C Patient Identification Group.

Margaret Meserve, MS, CGC is a genetic counselor at Boston Children’s Hospital in the Yu Lab. She has always been interested in ways of communicating complicated scientific concepts in accessible language and understanding how people perceive risk. With a background in neuroscience, genetics, and rare disease, she examines therapeutics and genetic concepts from both a scientific and psychological lens. Working in the Yu Lab, Margaret is responsible for evaluating variants and responding to interested physicians and families, as well as maintaining conversations and collaboration with the participants enrolled in studies at BCH. 

Lynn Bush, PhD, MSBioethics, MA, is a bioethicist, developmental scientist, and educator trained in Clinical Psychology, pediatric and neuropsychology subspecialty (doctorate and fellowships) and postgraduate training in neuroscience, fetology, and genomics. She is on faculty in Pediatrics division of Genetics and Genomics at Boston Children’s Hospital as well as faculty on Pediatrics at Harvard Medical School, HMS Genetics Training Program,  the HMS Center for Bioethics, and affiliate faculty RSZ Translational Neuroscience Center. Lynn is a member of the BCH Fetal Therapy Board and is Collaborator to the Yu lab on bioethical aspects of anti-sense oligonucleotide cases implemented and being considered. Historically, her clinical research, writing, and teaching focuses on ethical-psychosocial complexities and uncertainties raised by pediatric (ultra) rare diseases, neurogenetic conditions, and innovative translational interventions, for which she has leadership roles on national-international advisory committees and professional organizations. Currently, Lynn is Co-PI with Tim Yu, MD PhD and Ingrid Holm, MD MPH on an NHGRI funded study, Providing Ethical Guidance for the Development of Individualized Genomic Medicine as Rare as N-of-1.”



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