Rare Disease Research

11/28/2025

Wishing you a Thanksgiving filled with warmth, connection, and good food! 🦃This day is a beautiful reminder that our greatest treasures are the people we share our lives with. Take a moment today to truly appreciate your loved ones.

Wishing everyone a safe and joyful holiday! 🧡

11/21/2025

Our Atlanta team is looking for a Research Nurse 💜

Send your CV/Resume to:
Info@RareDiseaseResearch.com or visit our website for more information
www.RareDiseaseResearch.com

11/21/2025

Lennox-Gastaut Syndrome is a rare disease that affects 1 -3% of people with epilepsy. Rare Disease Research is committed to helping find novel treatments for those living with LGS.
Visit our website and see if one of our clinical trials is right for you
https://rarediseaseresearch.com/

11/13/2025

This past weekend, part of the RDR team had the opportunity to attend the annual FAST Global Science Summit in Orlando, FL.

This patient advocacy organization, dedicated to curing Angelman syndrome, brought together families from all over the world, scientists, and industry partners to showcase the latest resources and information available to those living with AS.

Each learning session was absolutely mind-blowing. The level of detail and insights into cutting-edge strategies for novel treatments were truly inspiring.

But the true highlight was the people. It was invigorating to connect with brilliant industry professionals and swap stories, challenges, and successes. A huge thank you to everyone who shared their expertise!

Most importantly, it was deeply moving and grounding to spend time with the families. Their courage and perspective are what truly drive our mission forward. Meeting you all was a powerful reminder of why we do what we do.

Time to put this knowledge and inspiration to good use! Thanks to the organizers for a fantastic event!

10/31/2025

On this magical Halloween 👻🎃, as ghosts👻 and goblins🧟 dance under the moonlight 🌖, our heart is filled with the warmest glow with endless blessings to be able share every day with such a fantastic team—true superheroes 💪🏼who bring hope and light to our patients and their families. These are all truly amazing, inspiring souls that make even the spookiest season feel like pure enchantment. 🎃🖤

09/07/2025

https://www.linkedin.com/posts/hanphanmd_patfurlong-luisaleal-sharonhesterlee-activity-7370521580055011328-b46L?utm_source=share&utm_medium=member_desktop&rcm=ACoAAAbVlpIBdx-O3RmFNbpAyiBoorlS5EN4tnk

On Duchenne Awareness Day, we unite to honor the remarkable individuals and families affected by Duchenne Muscular Dystrophy. Your courage and unbreakable spirit inspire us all. To those bravely facing Duchenne, your strength moves mountains each day. Families, caregivers, and advocates, your love l...

08/18/2025

Exciting update on cardiac safety in ! New data from Italfarmaco’s Phase 3 EPIDYS trial, presented at , shows givinostat led to less decline in heart function vs. placebo in ambulant boys with Duchenne, with no QTc prolongation. As a DMD cardiac expert, this reinforces givinostat’s potential to protect hearts amid progressive disease. Proud to highlight Dr. Han Phan as principal investigator for Italfarmaco in advancing this HDAC inhibitor. Full details: https://www.globenewswire.com/news-release/2025/07/11/3113828/0/en/Italfarmaco-Presents-New-Cardiac-Data-for-Givinostat-at-16th-European-Paediatric-Neurology-Society-EPNS-Congress.html?fbclid=IwdGRjcAMP80ZleHRuA2FlbQIxMQABHhYi-lG6DLGJvrDFI-VK9dptnV2v9bl0hEOm_lSaEQDmdgDfj1676o8nHTTB_aem_2981dGnzKPP0kzpTYZqy7A

In total, 11 abstracts on givinostat were accepted for presentation at the Congress, including two for an oral presentation MILAN, Italy, July...

08/12/2025

🎉 We’re Officially Open in New Jersey! 🎉

Rare Disease Research is proud to announce the grand opening of our newest clinical research site in Iselin, NJ!

Our NJ site is ready to welcome patients, collaborate with sponsors, and continue our commitment to innovation, patient-centered care, and advancing treatment options for rare diseases.

RDR–New Jersey: Because every trial is a step toward hope.

08/12/2025

We’re Officially Open in New Jersey! 🎉 Rare Disease Research is pleased to announce the grand opening of our newest clinical research site in Iselin, NJ, where we will collaborate with sponsors, welcome patients, and advance treatment options for rare diseases. Rare Disease Research

08/05/2025

A pivotal moment for RDR. Congratulations to Dr Renata Shih and the entire RDR research team to make this a success 🎉

Dosing the first patient in this clinical trial is a major milestone for the FSHD community. EPI-321 is the first therapeutic candidate designed to target the root cause of the disease. Gene silencing through epigenetic editing offers a potentially transformative approach to halting disease progression in FSHD,” said Dr. Shih - Principal Investigator at Rare Disease Research (RDR) in Atlanta.
This achievement reflects the outstanding teamwork and dedication of the entire RDR research staff and Epicrispr Biotechnologies team whose collaboration and commitment have been essential to advancing this important clinical trial.

We are deeply grateful to everyone involved for their invaluable contributions.

A pivotal moment for Congratulations to Renata Shih and the entire RDR research team to make this a success 🎉 Dosing the first patient in this clinical trial is a major milestone for the FSHD community. EPI-321 is the first therapeutic candidate designed to target the root cause of the disea...

07/29/2025

We’re expanding: Join us for the Grand Opening of RDR’s newest clinical research site in Iselin, NJ! 🎉

📍 When: Monday, August 11, 2025
🕑 Time: 2:00 PM – 5:00 PM ET

Meet Dr Ashutosh Kumar our Consulting Medical Director, triple board-certified in Pediatrics, Child Neurology & Epilepsy, and a leader in FDA-approved trials for Duchenne Muscular Dystrophy.

Explore our new space and connect with the team driving innovation in rare disease research.

Message us with any questions or to RSVP.