MLD announces plans for Rare Disease Day 2025

Med-Life Discoveries (MLD) has always made it a priority to recognize Rare Disease Day, using it as an opportunity to raise awareness about RCDP and other rare diseases. In the past our focus has been community-based awareness activities, but this year we have decided to change course and use the day as an opportunity to raise awareness about RCDP to US regulators.

We are excited to announce that the team from MLD will be traveling to Bethesda, Maryland this year to present at the FDA-NIH Rare Disease Day 2025 conference. This conference takes place at the National Institutes of Health (NIH) main campus on February 27-28, 2025 and brings together a wide audience including patients, advocates, researchers, healthcare professionals, industry representatives, and government staff.

To support MLD’s primary goal of raising awareness on RCDP, we will be presenting a poster entitled Voice of the patient in RCDP: The value of parents’ perspective in the development of therapy for an ultra-rare pediatric condition.

MLD and RhizoKids International have a long history of collaboration including the design of the Natural History Study and the development of an RCDP-specific quality of life scale (RhizoQoL). Despite the availability of natural history and quality of life data, the true burden of the disease on caregivers and their expectations for therapeutic improvements were still lacking. This lead to an Externally-Led Patient Focused Drug Development (EL-PFDD) session at the RhizoKids International family conference in 2024 and the development of a Voice of the Patient report (available soon).

“As a pediatric rare-disease company, we understand how critical it is that the voice of parents and caregivers are captured during the drug development process,” notes Shawn Ritchie, CEO and Chief Scientific Officer. “This presentation on Rare Disease Day, in-person in Bethesda, provides an excellent opportunity to tell the FDA about the complex and unrelenting nature of RCDP, the level of care required, and how committed the RCDP community is to find disease-modifying therapies that might provide even small improvements in physical or cognitive aspects of the disease.”

“The Patient-Focused Drug Development session was a truly powerful event following years of collaboration between MLD and RhizoKids that highlights the challenges and unmet medical needs of the RCDP community,” said Tara Smith, Executive Vice President. “I am honored to be able to present this information and give a voice to the RCDP community on Rare Disease Day.”