Rare Disease Research

05/20/2025

Today is recognized globally as Clinical Trials Day—but for us, every day reflects that mission.

At our clinic today, as on so many others, we are privileged to welcome patients from around the world—many of whom are living with rare diseases and have limited treatment options. Through their participation in our trials, they are not only accessing potentially life-changing therapies but also helping to drive scientific progress for future patients.

This is the heart of our work: expanding access to innovative clinical trials for those who need them most.

Thank you to our patients and their family for their continued dedication, compassion, and commitment, your efforts are deeply meaningful and truly impactful.

05/09/2025

Honor to be at the French Consulate Anne-Laure Desjonquères residence to learn about the NETVA program (New Technology Venture Accelerator) is a French government initiative designed to support early-stage deeptech startups in their international expansion, particularly into the U.S. market.

It is part of La Synapse, a broader strategy led by the Office for Science and Technology of the French Embassy in the United States.

Renata Shih MD, Han Phan, MD, Miro Pastrnak, PhD.

04/15/2025

Do you or someone you know have Prader-Willi Syndrome (PWS)? Rare Disease Research , LLC has recently begun a clinical trial sponsored by the The Foundation for Prader-Willi Research, and we’re open for enrollment!

Please visit https://lnkd.in/en2QCDRK to learn more about the eligibility criteria and contact our research team.

Intake Form: https://docs.google.com/forms/d/e/1FAIpQLScnzfDNsGMS1Fqa_7dRQtYU8OCeddLCO3CUtGkCdiGw4F0Rfg/viewform

This link will take you to a page that’s not on LinkedIn

04/12/2025

NOW ENROLLING: BEACH301 Clinical Trial
In partnership with Tyra Biosciences, Rare Disease Research (RDR) is committed to advancing research and providing access to a new clinical trial for eligible children with achondroplasia.

Key Details:
Study Name: BEACH301 Achondroplasia (NCT06842355)
Ages: Children with achondroplasia, ages 3 to 10 years old
Purpose: Explore the safety, tolerability, growth, and health outcomes of TYRA-300

04/08/2025

Rare Disease Research (RDR) & Gooseberry Research (GBR) are sponsoring the World Orphan Drug Congress (WODC) in Boston, MA, from April 22-24! 🌟

This event brings together industry leaders and innovators to shape the future of rare disease treatments. We’re excited to connect, collaborate, and explore new opportunities within the orphan drug space.

Visit us at Exhibit #439 to learn more about the groundbreaking work we’re doing and how we can drive progress in the rare disease community together.

04/07/2025

It’s time — SCRS Include Summit is here!

Rare Disease Research, LLC (RDR) and Gooseberry Research (GBR) are live at Exhibit #4 — come say hi, learn about our work in rare disease, and grab some goodies while you’re at it!

04/04/2025

LLC, we're proud to be leading the way in pioneering clinical trials across our locations in Georgia, North Carolina, and Florida. Our dedication to advancing research and providing access to hope for patients is stronger than ever. 🌟

We’re currently recruiting participants for trials focused on these rare diseases:

Facioscapulohumeral Muscular Dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), MCT8 Deficiency / Allan-Herndon-Dudley Syndrome (AHDS), Achondroplasia (ACH), Prader-Willi Syndrome (PWS), Myotonic Dystrophy (DM1), Pediatric Migraines, Angelman Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS), and Spinal Muscular Atrophy (SMA Type 3).

If you or someone you know might be eligible, please reach out using the contact info below-

Website - https://www.rarediseaseresearch.com/

Patient Intake Form -https://docs.google.com/forms/d/e/1FAIpQLScnzfDNsGMS1Fqa_7dRQtYU8OCeddLCO3CUtGkCdiGw4F0Rfg/viewform

04/01/2025

Now Enrolling: BEACH301 Clinical Trial

In partnership with Tyra Biosciences, Rare Disease Research (RDR) is committed to advancing research and providing access to a new clinical trial for eligible children with achondroplasia.

Key Details:
Study Name: BEACH301 (NCT06842355)
Ages: Children with achondroplasia, ages 3 to 10 years old
Purpose: Explore the safety, tolerability, growth, and health outcomes of TYRA-300

Learn more about achondroplasia at www.achondroplasia.bio. Contact advocacy@tyra.bio or visit achondroplasiatrial.bio to see if your child may qualify.

04/01/2025

So excited to introduce Dr. Scott Batchelor, MD, as the newest member of the LLC team! With 16 years of experience in pediatric emergency care at Children’s Healthcare of Atlanta and WellStar hospitals, Dr. Batchelor has treated over 100,000 patients annually in Level 1 and Level 2 trauma centers. His expertise in high-stakes, multidisciplinary care is exactly what we need to keep pushing for better treatments for rare diseases. Welcome to the team, Dr. Batchelor -we’re lucky to have you!

03/31/2025

March Madness fever hit Rare Disease Research – but instead of hoops, we battled it out in a Connect Four Tournament! 🏀🟡🔴 The competition was fierce, the strategy was real, and the friendly trash talk was next level. Who knew Connect Four could get this intense? 😆🏆

03/28/2025

We are excited to partner with Biosciences to bring the BEACH301 clinical trial for eligible children with achondroplasia to families in the southeastern US, starting in Atlanta. Stay tuned for details!

🌐 Visit at www.achondroplasia.bio to learn more about achondroplasia.

03/28/2025

We are excited to partner with Biosciences to bring the BEACH301 clinical trial for eligible children with achondroplasia to families in the southeastern US, starting in Atlanta. Stay tuned for details!

🌐 Visit at www.achondroplasia.bio to learn more about achondroplasia.

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