Sofia Sees Hope is proud to bring our web series “Let’s Chat About …” to the Leber congenital amaurosis and rare inherited retinal disease communities. We've developed the series with those living with LCA and IRDs in mind, but we invite all members of our community, including those in research, industry, and the regulatory communities to join any of the sessions, as we look ahead to a common goal of advancing treatments for rare retinal disease.
SEPTEMBER 29, 2021 - 1:00pm ET
Jill Dolgin, PharmaD
Executive Director, Global Patient Advocacy at AGTC
Let’s Chat About …The importance of the patient voice in rare disease
Jill Dolgin, PharmD is the Head of Patient Advocacy at Applied Genetic Technologies Corporation (AGTC) which is a clinical-stage biotechnology company developing transformational genetic therapies for inherited retinal diseases. Dr. Dolgin leads the patient and professional engagement strategy to drive disease awareness and clinical trial recruitment efforts for the AGTC pipeline. She is responsible for incorporating the voice of the patient throughout drug development and corporate culture. She has over 20 years of global biopharmaceutical experience in Medical Affairs, Corporate Communications, Patient and Professional Advocacy, and Public Policy.
Her role within the company is to ensure that the needs of the patients are considered and incorporated into every aspect of drug development. Externally, Jill works with patient advocacy groups to educate patients and families about the importance of participating in clinical trials, gene therapy and the importance of “getting your voices heard” to help consumers, healthcare professionals, and policy makers understand your challenges in living with your disease. Dr. Dolgin earned a Doctorate in Clinical Pharmacy from the University of the Sciences at Philadelphia and a Bachelor of Science degree in Pharmacy from The Ohio State University.
OCTOBER 18, 2021 - 1:00pm ET
Vice President, Clinical Development-Ophthalmology
Let’s Chat About … CRISPR and gene editing technology
Monica is the Vice President of Clinical Development in Ophthalmology at Editas Medicine.
Monica has focused her career on ophthalmic drug development. She has led teams through all phases of clinical development, regulatory approval, commercial launch, and life cycle management. Her expereince includes leading the development of ophthalmic gene therapy programs for age related macular degeneration and diabetic retinopathy, leading the successful approval of Xiidra for dry eye disease, and most recently leading gene editing programs in inherited retinal disease. She brings experience from various instututions, including The Wexner Medical Center, Alcon Novartis, Shire Pharmaceuticals, and Regenxbio.
Monica earned a Doctorate of Optometry, Master of Public Health in Epidemiology/Biostats, and a Bachelor of Science in Biology, all from The Ohio State University. She also completed a postdoctoral residency in Anterior Segment at The Ohio State University.
NOVEMBER 9, 2021 - 1:00pm ET
Daniel de Boer
Founder and Chief Executive Officer
Let’s Chat About … ProQR's work in treatments for inherited retinal disease
Daniel de Boer is Founder and Chief Executive Officer of ProQR, which was incorporated in 2012. Daniel is a serial entrepreneur and passionate advocate for rare disease patients. After one of his children was diagnosed with a rare disease, he started ProQR to develop RNA therapies for rare diseases. Under Daniel’s leadership, ProQR developed a platform that yielded a diversified pipeline of potential treatments for rare diseases and raised more than $400M in funding, including an IPO on Nasdaq. Before founding ProQR, Daniel was founder and Chief Executive Officer of several technology companies. Daniel is also co-founder and strategic advisor to Amylon Therapeutics and Wings Therapeutics, strategic advisor at Frame Therapeutics, Meatable, Algramo and a member of the advisory board at the Termeer Foundation. In 2018 Daniel was named "Emerging Entrepreneur of the Year" by EY. In 2019 Daniel was selected for the Young Global Leader program at the World Economic Forum.
Sofia Sees Hope is a 501c(3) nonprofit global patient advocacy organization dedicated to transforming the lives of those affected by blindness caused by rare inherited retinal diseases. Our mission is to generate awareness, raise funds for research, and provide outreach, support and education to those affected by LCA and other rare retinal diseases.
Leber congenital amaurosis (LCA) is a rare inherited retinal disease (IRD) that causes the degeneration of the cone and rod cells in the retina. It is characterized by severe vision loss at birth and results in complete blindness. As a rare disease that is not widely known or understood, LCA can often create feelings of isolation, frustration and helplessness.
All families affected by LCA can benefit from opportunities to connect and share information with other families, medical specialists and advocacy groups. A vital element of the Sofia Sees Hope mission is to provide more opportunities for all people affected by LCA to connect and provide mutual support and information. For more information about Sofia Sees Hope visit our website.
If you'd like to support Sofia Sees Hope and our mission to educate and connect the LCA and IRD communities, you can donate here.
This webinar series is made possible by
- Past Webinars -
JANUARY 27, 2021 - 1:00pm ET
Senior Director, Scientific Outreach & Community Engagement
Foundation Fighting Blindness
Let’s Chat About … What’s in the therapy pipeline for Leber congenital amaurosis.
For 16 years, Ben has been reporting on retinal research for all of FFB’s electronic and print publications. In addition, he presents the latest scientific advancements at local and national events for patients and families, and conducts various training activities for staff and constituents. He enjoys working with constituents one-on-one to help them understand their retinal disease and the research underway that may benefit them. Ben also leads the company’s outreach to eye care professionals throughout the United States.
Ben has written three books – Retina Boy, Jerry’s Vegan Women, and The Vegan Monologues – all published by Loyola University (Maryland). He earned a Master of Arts in writing from Johns Hopkins University, a Master of Science in systems management from the University of Maryland, and a Bachelor of Science in computer information science from Cleveland State University.
FEBRUARY 16, 2021 - 3:00pm ET
Wiley A. Chambers, MD
Supervisory Medical Officer
Office of New Drugs, Center for Drug Evaluation and Research
Food and Drug Administration (FDA)
Let’s Chat About … What it takes to receive approval for a new treatment for rare disease.
Wiley A. Chambers, MD, is a Supervisory Medical Officer in the Office of New Drugs, Center for Drug Evaluation and Research at the Food and Drug Administration (FDA). After receiving an undergraduate degree from Colgate University, Dr. Chambers completed medical school and a residency in Ophthalmology at The George Washington University School of Medicine and Health Sciences in Washington, DC. He is currently a Clinical Professor of Ophthalmology and Adjunct Assistant Professor of Computer Medicine at The George Washington University. He joined the FDA in 1987, as a primary reviewer for ophthalmic drug products and in 1990 became a Supervisory Medical Officer for Ophthalmologic Drug Products. In this capacity, Dr. Chambers has supervisory responsibility for the clinical review of ophthalmologic drug products and ophthalmic therapeutic biologic products submitted to the Center for Drug Evaluation and Research. Additionally, Dr. Chambers is the recipient of numerous Public Health Service, FDA and Center for Drug Evaluation and Research awards for his work with the FDA and he has served as the American Academy of Ophthalmology’s Delegate to past United States Pharmacopeia Conventions.
MARCH 15, 2021 - 1:00pm ET
Licensed Genetic Counselor and Research Study Coordinator
Massachusetts Eye and Ear
Let’s Chat About … The ins and outs of genetic testing.
Emily graduated from the University of St. Thomas with a Bachelor of Arts in Biology. She went on to earn her master’s degree in Human Genetics from Sarah Lawrence College. Prior to working at the Ocular Genomics Institute, she worked as a pediatric genetic counselor at The Children’s Hospital of Philadelphia.
Emily joined the OGI in 2011, where she provides risk assessment and genetic counseling services to families with inherited retinal dystrophies. She also serves as the study coordinator for the research study of molecular genetics of inherited retinal dystrophies.
APRIL 19, 2021 - 1:00pm ET
Jonathan Stokes, MBA
Director, Patient-Centered Outcomes Research
Let’s Chat About … Why natural history and patient outcome studies are important.
Jonathan has a devoted interest in understanding and bringing to light the patient voice and perspective, with over 16 years of research study design and implementation experience. His experience is primarily in health outcomes research; specifically, the development and evaluation of clinical outcomes of assessment (COAs) for use in clinical trials intended to substantiate product labeling goals, as well as use of COAs in real world clinical practice. Areas of focus include the evaluation of cardinal signs and symptoms of disease, health-related quality of life, improvements and activation in treatment adherence, understanding unmet need, and exploring the burden of disease. Jonathan holds an MBA from Northeastern University
MAY 13, 2021 - 1:00pm ET
Todd A. Durham, MS, PhD
Vice President, Clinical & Outcomes Research
Foundation Fighting Blindness
Let’s Chat About … My Retina Tracker
Todd Durham is the Vice President, Clinical & Outcomes Research at the Foundation Fighting Blindness, a national non-profit that funds research to treat and cure inherited retinal diseases. In his current role, Todd is responsible for directing the Foundation’s Clinical Consortium of retinal experts, developing strategies to enhance product development, partnering with industry, and providing technical input on partnered programs and investment decisions. Todd has over 25 years of drug development experience. Prior to his current position, he contributed to research on numerous marketed products as Director of Biostatistics with IQVIA’s Real World Evidence Solutions, was a doctoral fellow with Bristol Myers Squibb, and worked in various statistical and leadership roles for Novan, Inspire Pharmaceuticals, Quintiles, and as a self-employed consultant. Todd earned a BSPH and MS in biostatistics and a PhD in health policy and management (Decision Science and Outcomes Research) from the UNC Gillings School of Global Public Health.
JUNE 16, 2021 - 7:00pm ET
Panelists: Tami Morehouse and Jack McCormick
Let’s Chat About … the importance of self-advocacy
Tami Morehouse made research history when at age 44, she participated in a clinical trial for gene therapy for LCA2 (RPE65). Ultimately, Spark Therapeutics developed the drug that was marketed as LUXTURNA® following Federal Drug Administration approval in December 2017.
Tami volunteers in Sofia Sees Hope’s Family Connections program, which connects people within the LCA community to share information and provide support. She is also an SSH Ambassador. Our Ambassadors give encouragement to Leber congenital amaurosis (LCA) patients and caregivers, they attend conferences, keep up with research, and share their stories and experiences to help others.
Tami and her husband, Mike, who also is an Ambassador, live in Ashtabula, Ohio, and she works as an information and referral specialist for 211 of Ashtabula County. She and Mike represented SSH at the Foundation Fighting Blindness VISIONS 2018 conference in San Diego. She served as a panelist at our LCA Family Conferences in 2018 in Mystic, Conn., and 2019 in Philadelphia and privately talked with LCA patients and their families about frustrations with a lack of diagnosis, daily living, fears surrounding clinical trials and her experiences with them.
Jack McCormick of Ontario, Canada, graduated from Wilfrid Laurier University in Waterloo, Ontario, in 2018. Diagnosed with LCA2 due to a mutation in his RPE65 gene, Jack is a passionate advocate for inclusion and accessibility on all fronts. He initially tried to hide his blindness but that all ended when he got Jake, his beloved guide dog. In college he founded Eye To Eye, a student club whose goal is to illuminate stigma associated with vision impairments. He volunteers with Fighting Blindness Canada, including working on the 2018 Young Leaders Summit.
He is an organizational development consultant in the healthcare industry, is board vice chair of CNIB ON/QC and serves on the Metrolinx Accessibility Committee. In 2021, he launched the Lights with Jack McCormick podcast, what he describes as “my attempt to unlock the secrets to living a more meaningful and fulfilling life so that together we can find the lights in our life. Thank you for joining me as we connect with business leaders, personal development experts and inspiring people with stories to tell.”
He is also an SSH Ambassador, and writes a column in our quarterly newsletter about navigating adulthood with vision loss.