“Considering recent advancements in research, diagnosis, treatment options, and care, I imagine the future of those living with rare diseases to be promising, despite the enormous challenges that lie ahead. We have seen government agencies, industries, and patient advocacy groups share their commitment and make contributions to progress rare disease research, treatment, and access. It is gratifying to be part of Prothena and its mission to make a real impact for these patients and their caregivers through the development of our investigational drugs and medical information exchange.” – Lenh Mong, Regulatory Affairs

“I imagine a world with multiple treatment options to choose from that take into account the patient’s needs, quality of life and the trajectory of the disease. I imagine access to care and financial support for those in need. I hope those living with rare disease find comfort, encouragement and support in their communities. For physicians, I imagine confidence in recognizing, diagnosing and managing the disease. For the broader community, I hope for improved education and resources.” – Karthik Veera, Medical Affairs

“I imagine a future with faster, accurate diagnosis of rare diseases through enhanced public and medical community awareness, active engagement with patient advocacy networks and consortiums, and the integration of advanced technologies and diagnostic tools. Enhancing awareness provides patients with more access to appropriate medical care, thereby, enabling patients the opportunity to receive timely and effective treatment. Furthermore, the progression of clinical trials is expanding treatment options for unmet needs, offering clinicians and patients a broader range of choices in disease management and the opportunity for enhanced quality of life. I see a future of empowerment in the rare disease community.” – Jennifer Zimmerman, Global Site Management

“I imagine the future to be one of hope, equity, and progress. Every person, regardless of the rarity of their condition, should have access to life-changing and life-saving treatments. This means faster, more accurate diagnoses, access to effective treatments, and continued investment in research. Ultimately, the future I imagine is one where having a rare disease does not mean living without options.” – Preeti Bajaj, PhD, Global Health Economics & Outcomes