At ConSynance Therapeutics, our mission is to turn breakthrough science into transformative therapies for those facing rare central nervous system diseases, such as Prader-Willi Syndrome (PWS) and hypothalamic injury-induced obesity (HO). On this Rare Disease Day, we stand in solidarity with the rare disease community to shine a light on conditions that often go unnoticed.
Rare disease day 2024 falls on a uniquely symbolic date- February 29, the rarest day of the year. Despite affecting over 300 million people worldwide, rare diseases frequently receive less attention and research funding than more common conditions. This day serves as a reminder of the importance of inclusivity in healthcare and the need for continued research and development in the field of rare diseases.
Educating and Engaging
Rare diseases affect millions but often stay hidden in the shadows. Through dedicated education and awareness efforts, we can change that narrative. We encourage everyone to explore the various resources listed below, including recent FDA webinars on rare diseases. These materials offer valuable insights into the challenges faced by the rare disease community and highlight ways to engage and effect real change.
“Show your stripes”
The zebra, adopted as the official mascot for rare disease patients, symbolizes the importance of looking beyond the expected. The medical saying:
"when you hear hoofbeats, think horses, not zebras"
reminds medical professionals to consider a common diagnosis first. Yet, for those living with rare diseases, they embody the zebras—unique and deserving of recognition and understanding.
ConSynance Therapeutics: A Milestone to Celebrate
Our successful Phase 1 trial of CSTI-500 for Prader-Willi Syndrome (PWS) in 2023 was a significant milestone. Demonstrating a promising safety profile and pharmacokinetics in PWS patients, CSTI-500's progress brings some hope for these rare disease patients and their families. We are profoundly thankful to every trial participant, whose courage and commitment are instrumental in advancing the development of ground-breaking treatments. The participation in trials like ours provides hope for not only your future, but the entire rare disease community.
More information on PWS as well as HO is provided on our website here: The Rare Diseases
This Rare Disease Day, let’s amplify the voices of the rare disease community. Join us in this vital conversation. Spread awareness, support research, and embrace the power of community.
Together, we can make a difference.