Today: Rare Disease Day at Liberty Science Center

Today, Ovid Therapeutics, Marinus Pharmaceuticals and Ultragenyx Pharmaceutical are partnering with the Liberty Science Center (LSC) for the third annual Rare Disease Day. The free event is open to those impacted by rare diseases such as Angelman Syndrome and FragileX, along with their caregivers and close family members. There will be a number of activities for families including STEM programming and tours of LSC. Members of the National Organization for Rare Disorders (NORD) will also be in attendance.

In an exclusive interview between Patch.com and Ovid Therapeutics' chief medical officer Amit Rakhit, M.D., we learned more about what attendees can expect from today's Rare Disease Day event. including

PATCH: What can attendees expect from tomorrow's Rare Disease Day event?
Dr. Rakhit: The day is meant to celebrate the people and families who live with rare diseases everyday, and for the general public to learn more about the more than 7000 rare diseases that affect about 1 in 10 people around the world. We hope that the event is educational but also an opportunity to have fun and explore the great exhibits and science at Liberty Science Center.

PATCH: How many attendees do you anticipate having?
Dr. Rakhit: About 200 people have pre-registered for the event but we anticipate that number may change.

PATCH: How common are rare diseases, and who do they affect most?
Dr. Rakhit: Rare diseases affect everyone. There are more than 7000 rare diseases characterized by a diversity of disorders. A few facts:

• Over 7000 rare diseases are characterized by a broad diversity of disorders
• Rare diseases affect 1 in 10 people (>350 million people worldwide)
• 80% of rare diseases have identified genetic origins
• ~50% of the people affected by Rare Disease are children
• 30% of children with rare disease will not live to see their 5th birthday
• 95% of rare diseases have no FDA approved treatment or therapy.
• Delays in diagnosis (sometimes up to 8 years) and inability to access appropriate care are common
• High costs are becoming more of an issue for those who do have access

Source: Global Genes https://globalgenes.org/rare-facts/ ; NORD website;https://rareundiagnosed.org/rare-disease-facts/ and EUORDIS websitehttps://www.rarediseaseday.org/article/what-is-a-rare-disease; accessed 3/29/19

PATCH: Why are you helping advocate for this cause specifically?
Dr. Rakhit: Our mission at Ovid is to develop impactful medicines for patients and families living with rare diseases of the brain (neurology). Our commitment to our patient communities is about responsibility to seek innovation, to understand the challenges faced by patients and their families, to communicate with them clearly and openly, and ultimately to develop medicines that transform their lives. Anything else you'd like to share? We have a commitment to people- to people living with rare neurological diseases and their families and caregivers, to the scientific and medical community so we advance our approaches to research and development in rare brain diseases, to our investors and supporters, and to our employees and family. We look forward to making bold decisions to make a difference for patients.

Join in today!