Rare Disease Research

Our Atlanta location previously on Howell Mill Rd has recently moved to a brand new facility. Here is our new address.
1730 NE Expy NE, Atlanta, GA 30329

Apr 02,2022

ANNALS of Clinical and Translational Neurology

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy

Feb 15,2022

Recent Events

Fall 2021 - Winter 2022

Feb 14,2022

Our Newsletter - Winter 2022 | Vol. 5

The official Newsletter of Rare Disease Research, LLC

Feb 13,2022

Challenges in Designing Rare Disease Clinical Trials

Clinical News

Jan 04,2022

Genetic Disorders

What Causes Hereditary Diseases?

Nov 17,2021

Is your Child Developing Properly?

A Checklist that Helps Parents to Recognize the First Signs of Serious Neuromuscular Diseases.

Oct 05,2021

Recent Events

Summer - Fall 2021

Oct 04,2021

Our Newsletter - Fall 2021 | Vol. 4

The official Newsletter of Rare Disease Research, LLC

Jul 23,2021

Our Newsletter - Spring 2021 | Vol. 3

The official Newsletter of Rare Disease Research, LLC

Feb 25,2021

Our Newsletter - Winter 2021 | Vol. 2

The official Newsletter of Rare Disease Research, LLC

Feb 05,2021

Celebrating Rare Disease Day with Jett Foundation

Friday, February 26, 2021 | 2:00-3:30 PM ET

Feb 03,2021

Our Newsletter - Winter 2021 | Vol. 1

The official Newsletter of Rare Disease Research, LLC

Jan 22,2021

Support Local Artist Living with DMD

We invite you to support one of our patients with his fundraiser campaign.

Aug 18,2020

Clinical Study for patients with Metachromatic Leukodystrophy (MLD)

Clinical Study for patients with Tourette Syndrome available at Rare Disease Research.

Apr 29,2020

Supporting Our Community: PPMD

Make your gift today so that PPMD can maintain their commitment to research, care, and advocacy in the face of the most challenging time we’ve ever experienced.

Apr 13,2020

Supporting Our Community: Muscular Dystrophy Association

In the words of one of our patients, Ethan LyBrand, "Through the good times and bad, the Muscular Dystrophy Association helps kids like me."

Mar 17,2020

Safety Policy for Infection Control

RDR is closely monitoring the ongoing Coronavirus disease pandemic (2019).  As the current situation continues to change, our utmost priority is the health and safety of RDR staff, patients, and their families.

Mar 01,2020

Parent Project Muscular Dystrophy (PPMD)

Saturday, March 14, 2020 | Join for a FREE all-day meeting on Duchenne care, comprehensive research updates, and advocacy opportunities – breakfast and lunch are included!

Mar 01,2020

MG Walk Myasthenia Gravis Foundation Of America

Saturday, March 14, 2020 | Atlanta 2020 MG Walk. A world without MG. Come together to support the MG Community. Register to enter the fight against MG now!

About Rare Diseases

In the United States, a rare disease is defined as disease that affects less than 200,000 people.  With 7,000 known rare diseases affecting 30 million Americans and an estimated 350 million people worldwide.  Of those statistics, about 75% of rare diseases are children, and about 30% of patients with rare diseases will not live past the age of 5. 

In 1983, the FDA approved The Orphan Drug Act which gives incentives to companies that work to develop treatments for rare diseases. Since then, there have been nearly 350 treatments approved for various rare diseases.


Rare Diseases

30 Millions

Affected Americans


Approved Treatments


You or your child’s participation in our research is voluntary, and you are free to withdraw at any time. There will be no penalty or loss of benefits to which are you are otherwise entitled. We may be able to compensate you for your time and travel. Please do not hesitate to call us or send a message. We'll be happy to answer your questions and tell you more about our research.

Duchenne Muscular Dystrophy
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Spinal Muscular Atrophy (SMA)
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Pediatric Epilepsy
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Metachromatic Leukodystrophy
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Angelman Syndrome
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Fragile X Syndrome
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Becker Muscular Dystrophy
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CDKL5 Deficiency Disorder
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Excessive Daytime Sleepiness in Patients with Myotonic Dystrophy Type I
Learn more


Call our research team at: 678 - 883 - 6897
If you are experiencing an emergency, please call 911.


Please complete the form below to get more information about available research studies or
to schedule a call.

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