Rare Disease Research, LLC

Our team Edward C. Smith, MD, Anthony Giordano, MD, Renata Shih, MD & Alpa Khushalani just returned from the MDA 2025 Conference feeling inspired and energized. The event was filled with groundbreaking research, innovative ideas, and thought-provoking insights from industry leaders, clinicians, advocacy organizations. and families. Our booth attracted many like-minded professionals exchanging ideas and exploring collaborations that could accelerate research while incorporating the needs of the patient community.?A huge thank you to the organizers, speakers, and everyone who made this event a success. #MDA2025 #NeuromuscularResearch #PatientsFirst

We are excited to share that Rare Disease Research, LLC will be attending the SCRS IncluDE Site Solutions Summit? on April 7-8, 2025, in Atlanta, Georgia! This summit brings together clinical research leaders to foster collaboration and develop impactful solutions to ensure inclusive access to clinical trials. At RDR, we are committed to transforming the clinical research landscape by partnering with innovators to drive progress. The summit provides a unique opportunity to engage in interactive learning and open discussions focused on equitable trial access, partnerships, operations, and patient care. We look forward to connecting with industry peers, explore\ing collaborative opportunities, and advancing our shared mission to create meaningful change in clinical research. Be sure to stop by our exhibit booth #4! Let’s Partner for Progress and work together to shape the future of clinical research! #RareDiseaseResearch #SCRS2025 #ClinicalTrials #InnovativeSolutions #HealthcareInnovation #RDRLLC

?????? Celebrating Certified Nurses Day! At Rare Disease Research LLC, we’re lucky to work alongside some of the best Nurse Practitioners in the field — Emily Murray, Kallie Platt, and Meg Hudspeth! Their dedication to patient care and clinical research helps push forward new treatments for rare diseases every day. On Certified Nurses Day, we want to recognize their hard work, expertise, and compassion. The rare disease community is stronger because of healthcare professionals like them! Join us in celebrating Meg, Kallie, and Emily— drop a ?? in the comments to show your appreciation! #CertifiedNursesDay #ThankYouNurses #RareDiseaseResearch #NursePractitioners #ClinicalResearch #HealthcareHeroes

Welcome to the team, Austin Holland!

We’re honored to welcome Austin Holland as our new Clinical Site Manager at Rare Disease Research, LLC! With a strong background in clinical operations and leadership — most recently as a Senior Operations Manager — Austin brings the expertise and strategic mindset to drive our research initiatives forward. A Campbell University graduate and a dedicated runner, he embodies the perseverance and dedication that fuel our research. Welcome to the team, Austin! #WelcomeToTheTeam #ClinicalResearch #RareDiseaseResearch

Looking forward to an interactive Day 2 at the MDA conference! Gooseberry Research and Rare Disease Research, LLC are proud to sponsor the MDA Clinical & Scientific Conference (March 16-19, 2025) in Dallas, TX! Join us on March 18th from 7-8 a.m. CT at the Catalyst Pharmaceuticals Industry Forum, where Principal Investigator Edward Smith will be presenting. Looking forward to connecting and sharing our latest work in rare disease research as part of this exciting event! #MDAconference #RareDiseaseResearch #ClinicalTrials #Dallas2025

Connecting with industry leaders at MDA Conference to advance rare disease research. Join us at Booth # 525 to learn about our site network and innovative home health solutions. Welcome reception tonight at 6:00 PM EST. Let's discuss how we can collaborate. #RareDiseaseResearch #ClinicalTrials #GooseberryResearch #RDR #WelcomeReception #MDA

On Celebrate Scientists Day, we honor the brilliant minds whose discoveries drive progress in rare disease research. ???? At Gooseberry Research and Rare Disease Research, LLC our principal investigators express their deepest gratitude to the scientists whose groundbreaking work in gene therapy and rare diseases makes clinical trials possible. Their unwavering dedication to discovery is laying the foundation for life-changing treatments, and we are proud to support bringing their innovations to patients. In light of the recent federal cuts impacting scientific research, we stand in solidarity with all researchers, institutions, and communities working to ensure that progress continues. These challenges only strengthen our commitment to advancing science and improving the lives of those affected by rare diseases. The meticulous work of scientists worldwide is shaping a future filled with hope and breakthroughs. Join us in thanking them! Drop a ???? or ?? to show your support and stand with us in advocating for science.

Rare Disease Research, LLC is proud to welcome Dr. Renata Shih as our new Principal Investigator in Atlanta. A distinguished pediatric cardiologist, Dr. Shih brings over a decade of experience in patient care and clinical research, with a deep commitment to advancing treatments for rare diseases. Her impressive background includes a pediatric residency and fellowship at the University of Florida, a tenured professorship, and double board certifications in pediatrics and pediatric cardiology. Beyond her credentials, Dr. Shih is a dedicated advocate for patients and a leader in groundbreaking research. We are honored to have her expertise as we continue pushing the boundaries of pediatric clinical trials. Welcome to the team, Dr. Shih! hashtag #RareDiseaseResearch #PediatricCardiology #ClinicalTrials #Atlanta

In order to support several new gene therapy clinical trials, we are excited to announce that we have installed and certified a new compounding aseptic isolator at our Rare Disease Research, LLC site in Atlanta, Georgia! Her name is Gene-ifer. We are committed to delivering life-changing treatments for those with rare diseases, and that includes gene therapy treatments. Together, we're igniting hope and transforming lives! Join us on this remarkable journey! #RareDiseaseAwareness #PatientCare