2026 Speakers

Dr. Reimann is Co-Founder and Managing Partner of admedicum, where he helps biopharma companies integrate patient and caregiver perspectives into drug development.

Angi Robinson is Senior Vice President, Specialty Areas at Premier Research, where she brings more than 24 years of experience leading rare disease, pediatric, and other specialty clinical development programs globally.

Annie Kennedy is Chief Mission Officer at the RARE Foundation, where she advances evidence-based policy, advocacy, and patient-engagement initiatives to make rare diseases a national public health priority.

Barrett Tenbarge is a partner in Faegre Drinker’s FDA and regulatory group and a former Senate HELP Committee general counsel, advising life sciences clients on FDA regulation, compliance, product development, public policy, and congressional strategy.

Dr. Zhang is Associate Director of Patient-Centered Outcomes at Takeda and a former FDA scientist, specializing in clinical outcome assessments and patient-focused drug development.

Casey McPherson is the Founder & CEO of AlphaRose Therapeutics, a public-benefit biotech spun out from the To Cure A Rose Foundation, pioneering parent-led precision genomic medicine to develop ASO therapies, while leveraging equity crowdfunding to accelerate rare disease treatments.

Catherine Warren is Executive Director of the National PKU Alliance, where she leads efforts to advance PKU research, expand access to treatment and resources, and strengthen advocacy for individuals and families affected by phenylketonuria.

Dr. Ike is Head of Health Economics and Outcomes Research at Celcuity, with expertise in evidence generation, value strategy and oncology drug development.

Chris Garabedian is CEO of Xontogeny and Portfolio Manager of Perceptive Xontogeny Venture Fund, bringing deep rare disease and biotech operating experience from his leadership of Sarepta Therapeutics and his work backing early-stage life sciences companies.

Collen Caleshu is Senior Director of Translational Research at GeneDx and a clinician-scientist genetic counselor who works with biopharma partners to translate large-scale genomic, phenotypic, and longitudinal data into insights for rare-disease drug development.

Craig Lipset is an advisor, advocate, and educator in applied innovation for medicine developement, currently serving as Co-Chair of the Digital Trials & Research Alliance, as Clinical Innovation Lead for the Buffalo Initiative, and on the boards or advisory boards of organizations dedicated to transforming medicine development.

Dr. Grossman is Director of FDA's Division of Patient-Centered Development, where she leads initiatives that integrate patient perspectives into medical product development and regulatory decision-making.

Dr. Levy is Chief Medical Officer at Torque Bio, bringing extensive experience in rare-disease and genetic-medicine clinical development.

Danielle Dong is a rare disease medical affairs and scientific advocacy leader with global experience across patient care, research, and patient-centered evidence generation, currently serving as Scientific Advocacy Lead, Global Medical Affairs, Rare Disease at Sanofi.

Dennis Akkaya is Chief Commercial Officer at myTomorrows, where he leads the company’s global commercial strategy, accelerating patient access to treatments through clinical trial matching, pre-approval access, and stakeholder engagement in rare disease research.

Dr. Pichard, Chief Scientific and Medical Officer at the International Rett Syndrome Foundation, is a physician-scientist and rare disease parent-advocate advancing Rett syndrome therapeutic development, with prior rare disease leadership at NIH/NCATS.

Dr. Dashiell-Aje is Executive Director and Head of Patient-Centered Outcomes Science at BioMarin Pharmaceutical Inc., where she leads patient-focused evidence and clinical outcome assessment strategies across rare-disease development.

Dr. Smith is Medical Director and Principal Investigator at Rare Disease Research (RDR), and a pediatric neurologist specializing in rare neurological disorders.

Effie Parks is the host of Once Upon a Gene, a rare disease podcast sharing stories from families, advocates, clinicians, and researchers, and is a parent advocate whose work is rooted in her family’s experience with CTNNB1 syndrome.

Dr. Duke is Senior Vice President, Drug and Biological Therapies at Canal Row Advisors, a pediatric neuro-oncologist and former clinical reviewer in oncology who brings deep prior experience from FDA review of pediatric and rare cancer therapies.

Dr. Spies is Executive Scientific Director, Research and Strategy at Modus Outcomes, a THREAD company, specializing in patient-centered outcomes research and the integration of patient experience data and clinical outcome assessments into drug development.

Gemma Whiteley is Managing Director of FutureMeds @Home, where she leads the delivery of decentralized and home-based clinical trial services across Europe.

Dr. Phan is a board-certified pediatric neurologist, clinical investigator, and Founder and CEO of Rare Disease Research, with extensive experience leading clinical trials in rare neurological and neuromuscular diseases.

Holly Fernandez Lynch is an Associate Professor of Medical Ethics and Law at the University of Pennsylvania, whose work focuses on FDA policy, clinical research ethics, and access to investigational therapies.

Dr. Peay is a Senior Director at Faegre Drinker Consulting, specializing in patient-focused drug development, patient engagement, and bioethics research.

Drawing from her rare disease journey, previously captured in A Rare Journey, Hulda Björk Svansdóttir joins director Lainey Moseley for a live conversation on how mindset and purpose can help sustain joy and resilience, even when life feels difficult.

Dr. Caro is a globally recognized expert affiliated with LSE, McGill University, NUS, and Evidera known for pioneering innovations in health technology assessment, evidence integration, disease modeling, and healthcare value measurement.

Jake Caines is Chief Revenue Officer at Curant Health, where he leads commercial strategy and growth across employer, health plan, and life sciences markets, driving adoption of outcomes-based patient engagement solutions.

Dr. Berry is Chief, Division of ALS and Motor Neuron Diseases, Mass General Brigham, Associate Professor of Neurology at Harvard Medical School, and clinical researcher focused on ALS biomarkers and therapeutic development.

Jane Myles is Program Director at the Digital Trials & Research Alliance and a board leader at The Myositis Association, combining her drug-development experience with her lived experience of dermatomyositis.

Dr. Maynard directs FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, overseeing drug review across these areas.

Dr. Woodcock is a renowned former FDA Acting Commissioner and longtime Director of the Center for Drug Evaluation and Research (CDER) who shaped modern drug regulation over four decades, including leading therapeutics for Operation Warp Speed.

Dr. Khushalani is National Lead, HEOR – Epilepsy & Rare Syndromes at UCB, a physician-scientist focused on real-world evidence and evidence strategy in neurology and rare diseases.

Dr. McKenzie is a Senior Clinical Program Leader at Boehringer Ingelheim and pediatric nephrologist focused on advancing clinical development and pediatric trial design in kidney and rare diseases.

Jenn McNary is a nationally recognized rare-disease advocate, caregiver and co-founder of Canary Advisors who brings extensive experience in patient engagement, clinical-trial participation and Duchenne drug development.

Joe Walter is Senior Director of Marketing at Neurvati Neurosciences, where he supports market development and commercialization strategy for rare pediatric neurodevelopmental disorders and epilepsy.

Julia Taravella is Founder and Executive Director of the Rare Trait Hope Fund, where she created an ultra-rare AGU AAV9 gene therapy program from parent-led advocacy into an FDA-cleared clinical trial, bringing unusual firsthand experience in patient-led drug development

After her daughter Mila was diagnosed with a rare genetic disease, Julia Vitarello drove an unprecedented collaboration to develop the first-ever drug tailored to a single person, and now leads an international movement to advance individualized medicines.

Dr. Hoelzer is Senior Director of Patient Advocacy at the Biotechnology Innovation Organization (BIO), bringing deep expertise in rare disease policy, regulatory affairs, patient advocacy, and biomedical research, following her previous leadership role at NORD.

Dr. Healy is SVP of Regulatory Affairs at Entrada Therapeutics, where he leads global regulatory strategy across discovery and clinical development, drawing on extensive rare-disease experience and more than 30 formal interactions with FDA, EMA and other global health authorities.

Kim Quaintance-Lunn is Vice President, Head of Regulatory Science & Execution at Alexion, where she brings 30+ years of pharmaceutical and regulatory leadership experience across FDA, Eisai, Bayer, and Alexion to advance rare disease therapy development.

Kris O’Brien is Director Program Strategy Rare Diseases at ERGOMED, focused on rare disease and oncology trials, specializing in operational strategy, patient-centric design, and trial execution in complex studies.

Lainey Moseley is an award-winning producer, director, and rare disease advocate whose documentary RARE: A Rare Disease Revolution spotlights families navigating the race to develop treatments for ultra-rare conditions.

Lauren Morgenroth is the CEO and co-founder of TRiNDS, a CRO specializing in neuromuscular and rare disease clinical trials, with deep expertise in multicenter trial operations and patient-focused research.

Marc O'Connor is a principal owner and Chief Business Officer for Curant Health, and a nationally recognized thought leader on health care delivery, collaboration, and alignment.

Maria-Cruz Morillo, Global Head of Advanced Therapies, Rare Disease & CGT at Allucent, has over 25 years of experience in project management, strategy, and IMP supply across biotech and CRO environments, delivering strategic solutions for complex clinical trials.

Mark Stone is President and CEO of the FSHD Society and a longtime rare-disease advocacy executive who has built research and clinical-trial initiatives to accelerate therapies for patients and families.

Mary McGowan is President and CEO of the Foundation for Sarcoidosis Research, where she leads efforts to improve clinical trial access, accelerate drug development, and improve outcomes for people living with rare diseases.

Megan Freed is Director of Data Technology and Strategy at Parent Project Muscular Dystrophy (PPMD), where she leads data and registry initiatives supporting Duchenne research and care.

Micaela Rodine is Director of Clinical Operations at BridgeBio and a contributor to its achondroplasia clinical-development program, including the Phase 2/2b PROPEL Infant and Toddler study of infigratinib.

Dr. Binks is the Chief Medical Officer at Capricor Therapeutics, bringing over 25 years of experience in global clinical development and translational research, with a focus on immunology and rare diseases.

Michele Oshman is Chief Patient Advocate and Senior Vice President, Alliance Development at the Biotechnology Innovation Organization (BIO), where she leads patient advocacy efforts to elevate patient voice in biotech policy, partnerships, and access discussions.

Dr. Mellion is Chief Medical Officer at AstronauTx. She previously helped design and advance one of the earliest commercial rare disease platform trials in the UK.

Molly Painter, is Vice President of Sales & Strategic Accounts at Takeda, where she leads U.S. Plasma-Derived Therapies commercial strategy and brings senior leadership experience spanning rare disease, neuroscience, biotech launch, and patient/community engagement.

Dr. Knoble is a Senior Clinical Analyst with FDA’s Division of Rare Disease and Medical Genetics specializing in lysosomal diseases and advances patient-focused methodologies through the C-Path Rare Disease Clinical Outcome Assessment Consortium.

Dr. Kreher is the Chief Medical Officer at Alltrna and Board member at Rezolute Bio; she is a pediatric endocrinologist and has spent her entire career dedicated to rare disease drug development.

Nicole LaMarca is Executive Director of Scientific Engagement and Strategy in Patient Affairs at Sarepta Therapeutics and a pediatric nurse practitioner with more than a decade of experience caring for people affected by Duchenne muscular dystrophy and other rare diseases.

Paula Orandash is Director, Patient Engagement at ERGOMED, advancing patient-centered strategies in rare disease clinical trials, including community engagement, recruitment, and integrating the patient voice into study design.

Rachel Smith is Vice President and Global Head of Rare Disease at Parexel, providing expertise on effective, expedited, and robust clinical development strategies for rare therapies to accelerate these much-needed options to rare patients as safely as possible.

Dr. Pryluk is CEO and co-founder of PhaseV, a health-tech company developing causal machine learning–based platforms for adaptive clinical trial design and execution.

Dr. Huml is Executive Director, Therapeutic Strategy Lead, Rare Diseases at Worldwide Clinical Trials, bringing 30+ years of experience in clinical development, regulatory strategy, and patient-centered rare disease research.

Ron Bartek is Co-Founder and President of the Friedreich’s Ataxia Research Alliance (FARA), a nationally recognized rare disease advocacy leader whose work spans patient-led research, policy, regulatory engagement, and coalition-building across the rare disease ecosystem.

Dr. Chang is Medical Strategy Lead at UCB, where she works on the TK2 deficiency (TK2d) program, with a focus on earlier diagnosis and improving care pathways for patients with this ultra-rare mitochondrial disease.

Schiffon Wong, Founder of Schiffon Wong Strategic Advisory, helps biopharma leaders build evidence for payer reimbursement and patient access across neurology, immunology, and rare disease.

Sierra Phillips is the founder of the Warsaw Breakage Syndrome Foundation, an ultra-rare disease advocate and parent who brings experience in human genetics, clinical research, and business development.

Dr. Manton is CEO and Co-Founder of WinQure Medicine, where she is building a scalable platform to develop antisense oligonucleotide therapies for patients with ultra-rare genetic diseases.

Steven Grossman is President of HPS Group, former Executive Director of the Alliance for a Stronger FDA, and a longtime health policy leader who helped negotiate the Orphan Drug Act and Hatch-Waxman legislation.

Terri Klein is President and CEO of the National MPS Society and a longtime rare disease advocate with two decades of experience advancing research, patient support, and advocacy for MPS and related disorders.

Dr. Zhu is SVP, Head of Clinical Pharmacology, QSP & Toxicology at BioCryst Pharmaceuticals, with 25+ years of experience in quantitative and translational drug development.

Dr. Yan is Co-Founder, Director of the Center for Therapeutic Genetics at the Broad Institute of MIT and Harvard, Founding President of the N=1 Collaborative and a physician-scientist focused on advancing genetic medicines for patients with rare diseases underserved by traditional drug development.