Kara Baskin writes the award-winning Parenting Unfiltered newsletter and column for The Boston Globe. She also writes about food, quirky behavior, and real estate. She has reported and edited for Boston Magazine, The Boston Phoenix, New York Magazine, and The New Republic. Her book, Everything to Everybody: How to Wrangle Kids, Work, Aging Parents, and Spouses When You Just Want to Take A Nap (Grand Central Publishing), comes out in 2026. She grew up outside Boston and therefore maintains unbridled affection for Market Basket.

Dr. Chung is a clinical and molecular geneticist whose NIH-funded research spans autism, pulmonary hypertension, breast cancer, obesity, diabetes, and major birth defects. A national leader in the ethical, legal, and social implications of genomics, she has identified the genetic basis of more than 60 rare diseases. She directs the GUARDIAN study, which aims to expand newborn screening for genetic disorders and accelerate development of personalized therapies for affected children. Dr. Chung also leads major national collaborations to uncover the origins of autism, understand how it evolves across the lifespan, and improve supports for individuals and families. Her contributions have earned her the National Organization of Rare Disorders’ Rare Impact Award, and election to the National Academy of Medicine and the American Academy of Physicians. She holds a BA from Cornell, an MD from Cornell University Medical College, and a PhD in genetics from The Rockefeller University.

Dr. Steven Gray is a Professor in the Departments of Pediatrics, Molecular Biology, and Neurology at the University of Texas Southwestern Medical Center. Dr. Gray is the co-director of the UTSW Gene Therapy Program. Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system. His research focus has also been heavily focused in developing AAV-based treatments for neurological diseases, some of which have translated into clinical trials. He is inventor on over 25 awarded or pending patents and author on over 100 publications related to gene therapy.

Greg Huang is the Boston Globe's business editor. Prior to joining the Globe, he was an editor and reporter at Xconomy, New Scientist, and MIT's Technology Review. His writing has appeared in Wired, Nature, and other publications. He graduated from MIT with a PhD in electrical engineering and computer science.

Anna Kuchment is the health and medical editor at The Boston Globe. Before joining the Globe, she spent 14 years as a writer and editor at Newsweek, served as a senior editor at Scientific American, and was a science enterprise reporter at The Dallas Morning News. Her reporting has spanned COVID-19, climate change, health disparities, induced earthquakes, paleontology, concussions in rodeo cowboys, and more.

Anna holds a master’s degree from Columbia’s Graduate School of Journalism and was a 2021–22 KSJ Project Fellow at MIT. She is the author of The Forgotten Cure, a book on the modern race to develop phage therapy, and is completing Shaky Ground, co-written with historian Conevery Bolton Valencius, on human-induced earthquakes in energy-producing states. After years living elsewhere, she is thrilled to be back in her hometown, having grown up in Allston and Cambridge.

Shirley Leung is a business columnist, associate editor, and host of the Boston Globe Opinion podcast “Say More.” For more than a decade, she has written at the intersection of business, politics, gender, and workplace diversity. A weekly contributor to GBH’s “Boston Public Radio,” Shirley also serves on the board of the New England First Amendment Coalition. Her work has earned recognition from the Society for Advancing Business Editing and Writing, and she has been named among Boston Magazine’s “100 Most Influential People.” A four-time finalist for the Gerald Loeb Award for Commentary, Shirley previously served as the Globe’s business editor and interim editorial page editor. Before joining the Globe, she reported for The Wall Street Journal and began her career at The Baltimore Sun. She is a graduate of Princeton University.

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Born and raised in Weymouth, Senator O’Connor grew up in a close-knit South Shore community he has proudly represented in the Massachusetts Senate for nearly a decade. Patrick graduated from Weymouth High School in 2003 and attended Kingston University in London. After returning home, he ran for Weymouth Town Council on a platform of positive change and, at age 21, became one of the youngest elected officials in the nation. He later served as Town Council President. Throughout his time in office, Patrick has championed legislation that centers around protection of the vulnerable, including those with rare diseases. Particularly, he has filed this legislative session S.1602, An Act improving pediatric cancer research.

Sarah Rahal is a health and science reporter on the Globe’s Metro desk, covering public health issues with a particular focus on underserved communities across New England. She joined the Globe in 2025 after eight years at The Detroit News, where she reported on breaking news, local government, the COVID-19 pandemic, caregiving, and Detroit’s post-bankruptcy recovery. Her 2023 investigation into kinship caregivers revealed major gaps in Michigan’s support systems, spurred legislative change, and earned the state’s top enterprise reporting award.

A Knight-Wallace Reporting Fellow, Sarah has long centered immigration and health policy disparities in her work. She is a graduate of Wayne State University’s Journalism for Media Diversity program and a former president of AAJA Michigan. Born and raised in Dearborn, she is the daughter of Lebanese immigrants and a co-founder of a still-operating wholesale falafel business.

Marin Wolf covers the business of health care for the Boston Globe. Before her move to Boston, she was a health care reporter at The Dallas Morning News, where she wrote about public health, biotechnology and Texas hospitals. She led the paper's COVID-19 and reproductive care coverage. Her work has also appeared in Bloomberg News and publications across North Carolina. She is currently pursuing a master of public health degree from the Harvard Chan School of Public Health. A North Carolina native, she graduated from the University of North Carolina at Chapel Hill.

Join Say More for a live podcast conversation with Dr. Wendy Chung, Chief of Pediatrics at Boston Children’s Hospital, exploring the impact of the Children’s Rare Disease Collaborative (CRDC). Launched in 2018 and powered by 100+ physician-scientists and patient families, the CRDC is uncovering genetic causes of rare diseases and accelerating research-informed, precision care. Dr. Chung will share how this Boston-based collaboration is shaping national breakthroughs in diagnosis, treatment, and hope for families.

Boston, Silicon Valley, and Dallas are among the regions bringing unique strengths to rare disease innovation. What distinguishes each region’s approach to research, collaboration, and commercialization – and where do their paths converge? Hear from leaders on how cross-regional partnerships can accelerate breakthroughs for patients everywhere.

Join Boston Globe parenting columnist Kara Baskin for a candid conversation with Dr. Tommy Martin, a Med-Peds physician and popular medical/parenting creator whose son lives with Lamb-Shaffer syndrome. Dr. Martin brings the rare combination of clinical expertise and personal caregiver experience. Together, they’ll explore the challenges families face navigating rare disease, the role of storytelling and social media in building awareness, and how clinicians and caregivers can better work together to improve outcomes.

Meet the next generation of advocates driving progress in the rare disease community. From awareness campaigns to research collaborations, these young changemakers are reimagining what advocacy and leadership look like. Hear their stories and the bold ideas shaping the future of rare disease engagement.

Parents and caregivers are often thrust into the rare disease world without a roadmap, yet many emerge as some of the field’s most powerful advocates for change. This panel centers parents and caregivers as critical partners in advancing rare disease solutions, alongside political figures working to turn urgency into action. Spanning early diagnosis through long-term caregiving and advocacy, the discussion examines how parent-led efforts are influencing funding, legislation, and systemic change.

Anne O'Donnell-Luria is co-director of the Center for Mendelian Genomics at the Broad Institute of MIT and Harvard, where she is an Institute Member. She is also an Associate Professor at Boston Children's Hospital, Harvard Medical School. Her laboratory focuses on using genomic and transcriptomic approaches to increase the rate of rare disease diagnosis through improving rare variant interpretation, understanding incomplete penetrance, and empowering the discovery of novel disease-gene relationships. She is particularly interested in how we can leverage gnomAD, a massive reference population database, in these efforts. Dr. O’Donnell-Luria is also a practicing clinician at Boston Children’s Hospital (BCH), where she runs a clinic focused on evaluating families with epigenomic disorders. 

Daria Dragicevic is a rare disease patient, advocate, and researcher. Diagnosed with a nano-rare neurological disorder, DYT-27, which affects fewer than seven individuals worldwide, she transitioned her career toward researching speech and voice outcomes in people with neurological disorders. She is currently a PhD candidate in Speech, Language, and Hearing Sciences at Boston University. In parallel with her research, Daria is actively engaged in patient advocacy, working to elevate patient voices and promote increased funding, awareness, and equity in rare disease research. She is passionate about accelerating the development of effective therapeutics for those living with rare diseases.

Guadalupe Hayes-Mota is Director of Bioethics at the Markkula Center for Applied Ethics and a Senior Lecturer at the Massachusetts Institute of Technology, where he teaches biotechnology, artificial intelligence, and healthcare operations. A globally recognized executive and scholar, he bridges technology, policy, and ethics to advance equitable access to medicine. He founded Healr Solutions, an AI-driven company that optimized drug distribution in 43 countries, and held leadership roles at Biogen, Amgen, and GSK. Appointed to the NIH Cures Acceleration Network Board, he advises the European Commission on AI in healthcare, publishes in Forbes and Fast Company, and holds degrees from MIT and Harvard.

Sydney is a senior at Harvard studying chemistry and neuroscience. He first gained interest in the field of rare diseases as one of the youngest interns at Vigil Neuro, a biotech company developing therapies for rare brain disorders. After learning more about barriers to care and clinical uncertainty within this landscape, Sydney conducted health services research at MGH and co-authored a review paper and presented a poster on interventions to improve quality of care. He is also conducting research at the Bragg Lab in the Department of Neurology, MGH, where he studies X-linked dystonia parkinsonism. As president of Harvard’s Biotech Club, he also co-hosted Harvard’s first Rare Disease Hackathon in collaboration with the National Organization for Rare Disorders.

Jayme Betts is the mother of a seven-year-old daughter with Sotos Syndrome and an advocate for individuals with disabilities and rare diseases. Driven by her family’s experience navigating gaps in care and access, Jayme has worked to raise awareness and advance policy in Massachusetts, including founding the state chapter of the Changing Spaces campaign to promote adult-sized changing tables in public spaces. She serves on the board of the Arc of Massachusetts and is spearheading efforts to establish a dedicated Sotos Syndrome Clinic at Massachusetts General Hospital. Jayme lives in Weston with her husband and two daughters and is a Managing Director and Global Head of Front End Trading at BMO Capital Markets.

Jayme Betts is the mother of a seven-year-old daughter with Sotos Syndrome and an advocate for individuals with disabilities and rare diseases. Driven by her family’s experience navigating gaps in care and access, Jayme has worked to raise awareness and advance policy in Massachusetts, including founding the state chapter of the Changing Spaces campaign to promote adult-sized changing tables in public spaces. She serves on the board of the Arc of Massachusetts and is spearheading efforts to establish a dedicated Sotos Syndrome Clinic at Massachusetts General Hospital. Jayme lives in Weston with her husband and two daughters and is a Managing Director and Global Head of Front End Trading at BMO Capital Markets.

In-person guests are invited to enjoy a complimentary lunch in Studio B at Big Night Live, included with their event registration. During this time, the stream will pause for a break for all virtual attendees.

Brief Opening Remarks Provided by Rocket Pharma

Jazmin Aguilera is a host and senior audio producer for the Boston Globe. Previously the head of audio at Los Angeles Times, Jazmin has a long tenure in the audio industry spanning more than a decade. She was host and supervising producer for The Cut podcast where she produced irreverent audio essays on cultural hot button topics. She was a senior producer and interim executive producer at Conde Nast, producing for Vogue and Pitchfork as well as overseeing audio products for Wired Magazine. She was the inaugural producer fellow with the New York Times working on The Daily where she produced impactful news podcast episodes while simultaneously becoming an International Women's Media Fund grant recipient to report on the Mexican-Guatemalan border. She spent 5 years at the NPR show Snap Judgment, where she produced award winning first person feature episodes. She has won several accolades including Spotify's best podcast episode of the year 2019, Indiewire's second best podcast episode of the year 2019, Third Coast Audio Festival award Gold 2018 and Silver 2019. She and her team produced the podcast episode The Leaked TapeThat Upended L.A. Politics included in the Los Angeles Times 2023 Pulitzer Prize win in breaking news.

Deb is an active philanthropist on the board of Mackenzie’s Mission, an advisory board member for TIFF Advisory Services, and on the Dean’s advisory board for the Boston University School of Medicine. In the past she has served as board chair for MentorPrize, a member of the Advisory Board for Northeastern University’s venture accelerator IDEA, a Benefit Plan Investment Committee member for the American Red Cross; and a member of the Board of Managers for Strategic Investment Group. For over 30 years she was an executive in the institutional investment management industry focused on endowments, foundations, and pension plans. She co-authored pieces in the Journal of Cardiac Failure and MedEdPublish, and the book Expert Systems for the Technical Professional (Wiley & Sons, Inc.). A new resident to the Boston area, Deb enjoys travel, tennis, and being with her family.

Mackenzie, an AL amyloidosis patient, founded Mackenzie’s Mission in 2017, a non-profit focused on raising awareness and supporting research for amyloidosis. Educational initiatives include the Amyloidosis Speakers Bureau which has given over 475 presentations to more than 25,000 future and current medical providers, and a podcast titled “All Things Amyloid.” Additionally, over $1,875,000 has been donated to
advancing research on amyloidosis. She completed her education at Northeastern University and now works as a Physician Assistant at a local hospital. She has co-authored publications in the Journal of Cardiac Failure, MedEdPublish, and the Journal of Global Public Health. When not working, she enjoys traveling the world, tennis, and being with her family.

Join Boston Globe parenting columnist Kara Baskin for a candid conversation with Dr. Tommy Martin, a Med-Peds physician and popular medical/parenting creator whose son lives with Lamb-Shaffer syndrome. Dr. Martin brings the rare combination of clinical expertise and personal caregiver experience. Together, they’ll explore the challenges families face navigating rare disease, the role of storytelling and social media in building awareness, and how clinicians and caregivers can better work together to improve outcomes.

Bobby graduated from UMASS Boston in May 2024 with a bachelor’s in management and a concentration in Finance. He currently resides in Dedham, MA.

Bob Coughlin is a Managing Director at JLL and is the New England’s Life
Science and Healthcare Practice Group lead. He specializes in the representation of lab, GMP manufacturing and technology space. Bob most recently operated as the President & CEO of the Massachusetts Biotechnology Council, where his mission was to advance Massachusetts's leadership in the life sciences to grow the industry. Before joining MassBio, he served as the Undersecretary of Economic Development within Governor Deval Patrick’s administration, where he
prioritized both healthcare and economic development issues and was
a strong advocate for the life sciences industry in Massachusetts. Prior
to that, he was elected as State Representative to the 11th Norfolk
district for three terms.

Join Boston Globe parenting columnist Kara Baskin for a candid conversation with Dr. Tommy Martin, a Med-Peds physician and popular medical/parenting creator whose son lives with Lamb-Shaffer syndrome. Dr. Martin brings the rare combination of clinical expertise and personal caregiver experience. Together, they’ll explore the challenges families face navigating rare disease, the role of storytelling and social media in building awareness, and how clinicians and caregivers can better work together to improve outcomes.

Many patients, caregivers and professionals in the rare disease community often carry their personal experiences into their work, influencing how they approach care, advocacy, research and industry. This panel centers on shared lived experience as a catalyst for purpose and action across the rare disease ecosystem. Featuring perspectives from a patient turned healthcare provider, a parent and nonprofit leader, and an industry executive and caregiver, panelists will share how lived experience has shaped their professional paths. Through personal stories, the discussion will explore where the system continues to fall short and what must change to create a more connected and responsive future for rare disease patients and their families.

Join Boston Globe parenting columnist Kara Baskin for a candid conversation with Dr. Tommy Martin, a Med-Peds physician and popular medical/parenting creator whose son lives with Lamb-Shaffer syndrome. Dr. Martin brings the rare combination of clinical expertise and personal caregiver experience. Together, they’ll explore the challenges families face navigating rare disease, the role of storytelling and social media in building awareness, and how clinicians and caregivers can better work together to improve outcomes.

The panel will focus on how individuals living with rare skeletal conditions — and parents and caregivers supporting children with these conditions — navigate shared decision-making in ways that support overall health and well-being. With a focus on real conversations happening across the community, the discussion will underscore the importance of support networks when families face unfamiliar decisions.

Kristen DeAndrade is a relentless advocate, breaking down stereotypes and barriers surrounding skeletal dysplasia and disability. In 2024, she founded The Little Legs Big Heart Foundation, a nonprofit focused on advocacy, medical support and community empowerment for the skeletal dysplasia community. As the author of Little Legs, Big Heart, Kristen shares her journey of resilience, vulnerability and triumph, encouraging others to embrace their strength unapologetically. Based in West Palm Beach, Florida, she amplifies her voice through her foundation, writing and motivational speaking to drive meaningful change and foster inclusivity.

Olaide Adekanbi is a Scientist, Innovator, and Advocate working at the intersection of clinical research and rare disease policy. A graduate of Northeastern University, she has contributed to research at Harvard, UMass, and industry leaders including GSK, Sanofi, and bluebird bio. Currently, Olaide serves on the Massachusetts Rare Disease Advisory
Council, advising state leadership on policy impacting the rare community, and is an Ambassador for the Massachusetts Sickle Cell Association, where she founded the Young Adult Community Network. As Founder and CEO of SoundRest, she is developing a non-opioid, ultrasound-based therapy for Sickle Cell Disease pain. By integrating scientific rigor with state level policy and community leadership, Olaide is establishing a new model of patient-led innovation where lived experience is a primary catalyst for scientific discovery.

Gianluca Pirozzi, MD PhD, is Senior Vice President, Head of Development, Regulatory and Safety at Alexion, AstraZeneca Rare Disease. Dr. Pirozzi has over two decades of experience in drug development with a focus on immunology and rare disease. Previously, he served in various roles at Sanofi Genzyme, including Head of Development for Rare Diseases and Gene Therapy. He served on the Board of Directors of Imbria Pharmaceuticals for two years and is currently a Board Member of Timber Pharmaceuticals as well as scientific advisor for the Smith-Magenis Syndrome Research Foundation. With over 50 publications, Gianluca holds an MD degree from University Campus Bio-Medico and a PhD in Immunology from Sapienza University, both in Rome, Italy, and completed a Post Doctoral Research Fellowship in Immunology at the Pasteur Institute in Paris, France.

Meryl is the founder of the Adelia the Wonder Girl Foundation. Her work focuses on advocating for brain cancer awareness and improving funding for research at the local, state, and national levels. Since 2023, Meryl and her team have raised close to $700k, primarily through the Falmouth Road Race. These funds have been directed to the Brain Tumor Center that merges Dana Farber and Boston Children's Hospital. These funds are helping to propel research forward in order to find better treatments for brain cancer, but also to support families through this horrendous journey. Her fierce passion for improving outcomes for aggressive pediatric brain cancer is fueled by her daughter Adelia's passing at the age of 8 after battling Diffuse Midline Glioma for 15 months. The treatment protocol for this cancer has not changed since 1963, which is 6 weeks of palliative radiation, and Meryl is determined to move this needle in her lifetime.

Adelia's spirit continues to live on in every aspect of Meryl's life. Adelia was a talented violinist, and Meryl is a professional bassoonist that performs regularly throughout the Boston area. Meryl is also an Occupational Therapist that specializes in hand therapy. Adelia was kind, gentle, caring and giving, and Meryl carries her soul through all of the work that she does. Meryl enjoys being a mom to her two other children, and training to run the Boston Marathon for the Brain Tumor Center in April.

Scott Weintraub is Senior Vice President, US Business, Alexion, AstraZeneca Rare Disease, overseeing more than 1,000 colleagues across commercial, medical, operations, and support functions. He is responsible for the business’s reputation, customer satisfaction, revenue, and profitability, and also serves as site lead for the Rare Disease Headquarters in Boston. Scott brings over 20 years of biopharmaceutical experience in commercial strategy, marketing, and business development, having held senior leadership roles at Alexion since 2019.
He holds a BS in Finance and Marketing from Florida State University and an MBA in Health Administration and Policy from the University of Miami.

Becky Jackson Curran is a recognized disability inclusion advocate, professional speaker and media consultant dedicated to advancing equitable and authentic representation of people with disabilities. Born with achondroplasia, a rare genetic condition, she brings both lived experience and industry expertise to her work across corporate, entertainment and nonprofit sectors. Becky’s career includes roles with Creative Artists Agency, CBS Television Studios and the Equity & Inclusion department at SAG-AFTRA. She is the founder of DisABILITY In Media and serves on several national advisory boards. Becky has delivered more than 650 speeches, including two TEDx Talks, and continues to champion a more inclusive world.

Cristian Garcia is a family content creator and entrepreneur with millions of followers across social media. He is the founder of Thee Garcia Family and the clothing brand Puro Pa’ Delante, where he shares stories and videos centered on faith, family and resilience. Cristian uses his platform to inspire others through authentic storytelling, advocacy and entrepreneurship.

Jennifer Arnold, MD, MSc is a pediatrician, neonatologist, and expert in healthcare simulation at Boston Children’s Hospital. Her unique perspective on medicine has been shaped by the various ways she has engaged with the field: as a pediatric and adult patient, cancer survivor, mother, and doctor. Dr. Arnold is an internationally known speaker, published NYT best-selling author (Life is Short, No Pun Intended and Think Big), and recipient of awards for innovation in medical education and patient-care. She has spoken both nationally and internationally on healthcare simulation and her journey to become a physician as a woman has made inspirational speeches on overcoming obstacles as an individual with a disability.

Dr. Jennifer Kelly is Director of the Metabolic Bone Program and Chief of the Division of Endocrinology and Diabetes at University of Vermont Health, and a Professor of Medicine at the Larner College of Medicine. She has more than 20 years of experience caring for patients with metabolic bone disorders, including hypoparathyroidism, and is widely recognized for her leadership in clinician education in this field. Dr. Kelly served on the writing committee for the American Association of Clinical Endocrinologists’ 2020 Guidelines on Postmenopausal Osteoporosis and participates in multiple regional, national, and international committees focused on metabolic bone health. She previously chaired the International Society for Clinical Densitometry annual meetings and is the first Bone Health & Osteoporosis Foundation ambassador from the state of Vermont.

Patty is the Executive Director of the HypoPARAthyroidism Association, where she brings a powerful combination of professional leadership experience and personal passion for patient advocacy. A post-surgical hypoPARA patient since 2015, Patty understands firsthand the challenges of living with a rare disease. Her commitment to helping others navigate those challenges led her to volunteer with the Association, later joining the Board and serving as Chairman beginning in 2022. In February 2024, she stepped into the Executive Director role with a mission to strategically grow the Association, reach more patients and clinicians, and amplify the voice of the hypoPARA community.

Before dedicating herself to nonprofit leadership, Patty spent over 30 years in senior roles within the beauty industry, overseeing operations for up to 400 hair salon locations. She was a key member of Ratner Companies' senior field leadership team, recognized for delivering top-line results, launching new markets, and leading high-performing regional teams. Patty developed and implemented training programs, led initiatives to boost sales and profitability, and facilitated leadership onboarding programs that supported company growth and culture.

Known for her people-first leadership and relentless dedication to excellence, Patty has a passion for empowering others—whether in the boardroom or through patient support and education.

Outside of her professional life, she is wife to Bill for over 40 years, proud mom to Billy, and is a "Nina" to two wonderful grandsons. She loves spending time with family and friends, traveling, and enjoying Broadway shows

With over 20 years in healthcare — from laboratory services and phlebotomy to pharmacy compounding and nonprofit leadership — Michele’s career has been driven by one goal: improving patient lives. She currently serves as Associate Director of the HypoPARAthyroidism Association, where she leads advocacy, education, and public policy initiatives for the rare disease community.

Alongside her nonprofit work, she is a Certified Pharmacy Technician and Compounding Technician, and an active member of the Texas Pharmacy Association’s Public Policy Council. She is also a volunteer with Patients Rising, the EveryLife Foundation for Rare Diseases, and the Chronic Disease Coalition to elevate patient voices and influence healthcare policy.

A post-surgical hypoPARA patient since 1994, Michele brings lived experience and a deep commitment to patient advocacy, public policy, and rare disease education. She believes meaningful change happens when experience, empathy, and action come together — and is passionate about turning patient stories into policy solutions.

Dr. Zeinab Choucair is Vice President and Head of US Medical Affairs in Endocrine Medical Sciences, bringing a unique blend of clinical expertise, scientific leadership, and strategic vision to the advancement of innovative therapies. She has extensive experience across endocrinology, women’s and men’s health, regenerative medicine, orthopedics, and aesthetics, with a strong track record of translating emerging science and cell-based therapeutic approaches into clinical practice.

Dr. Choucair has led US and global medical affairs strategy, KOL engagement, and cross-functional initiatives supporting product development and lifecycle management, while also overseeing pharmacovigilance and drug safety operations. Her career spans leadership roles across multiple global and emerging healthcare organizations, where she has consistently driven evidence-based innovation and strengthened collaboration among healthcare providers, regulators, industry partners, and professional societies.

She is deeply committed to empowering patients and healthcare professionals through education and to translating science, medicine, and technology into meaningful clinical impact.

JoAnne was a former small business owner who provided website development services before surgery led to a diagnosis of hypoparathyroidism in 2015. Since then, she has focused on managing her health while becoming a patient advocate, building meaningful connections and lasting friendships within the hypoparathyroidism community. A lifelong New Hampshire resident, she enjoys spending time outdoors and loves hiking and snowmobiling when she’s able. She is the proud mother of two daughters and grandmother to a beautiful one-year-old granddaughter who brings endless joy to her life.

As the Director of Patient Advocacy at Rocket Pharmaceuticals, Amy strives to ensure that patients are always our North Star. Amy’s academic and professional background in community psychology and public health translated naturally into her work in patient engagement and advocacy following a rare disease diagnosis in her children. With over 16 years of experience in the space within patient organizations, industry consulting, and industry roles, she is dedicated to ensuring the perspectives and experiences of those impacted by rare disease as patients, caregivers and carriers, are included throughout the drug development process. She holds a Masters in Community Psychology and resides in Denver, Colorado with her family.