The Rare Roundup

Fall 2021 | Vol. 4

The official Newsletter of Rare Disease Research, LLC

"Coming together is a beginning, staying together is progress, and working together is success."

- Henry Ford

We are the dream team!

We made it! After the big move at the end of May and a few hectic months of adjusting, we have now settled in and our staff is enjoying the new space. Looking back, we were totally amazed at how we were able to conduct all study activities at our previous site! What can we say? We are the dream team and we always work together to overcome any challenges that come our way!

RDR Staffing News

It is very exciting to see how our team has grown over such a short period of time! We have recently welcomed the following new staff members:

  • Brenda Almaras, Clinical Research Assistant/Regulatory Specialist: Brenda has a Bachelor's degree in Psychology from Kennesaw State University and is currently working on her Bachelor’s in Biology. She brings experience from the Kennesaw Social Cognition and Interpersonal Violence research groups, and she is eager to learn and help the regulatory team at RDR!
  • Dillon Dang, Clinical Research Assistant: A medical assistant and recent graduate of the University of South Florida, Dillon moved to Georgia for the chance to work with Dr. Phan and the rest of the Rare Disease Research team. Dillon plans to attend medical school in the near future. He is a welcome addition to the team, and we look forward to showing him how Georgia does research. 
  •  Noella Skair, Certified Phlebotomist/Laboratory Specialist: Noella has come to RDR from Cohen Children’s Medical Center in New York. She brings a great sense of humor and an intense appreciation for chicken from Popeye's to RDR, in addition to her wealth of experience as a laboratory and surgical technician.
  • Meg Hudspeth, Nurse Practitioner: Meg has come to RDR from Children's Healthcare of Atlanta to give Lindsay, our once lonely nurse practitioner, a much needed hand. Meg obtained her Doctorate of Nursing Practice from Augusta University and brings extensive knowledge of pediatric care to the clinic. Welcome, Meg!
  • Jennifer Griffin, Bookkeeper/Regulatory Assistant: Coming from a background in security, Jennifer is our first Bookkeeper. She has been a valuable asset to the team helping organize our books and keeping the teams’ spending in check!
  • Olga Tenyuta, Budget & Contracts Specialist: Olga recently came to RDR from Russia, and we are so happy to have her! She served as the CFO of a large company in Russia, overseeing all financial analysis and reporting, and now brings all of this experience to RDR. Olga is very enthusiastic to learn new things, and we are equally as enthusiastic to have her on board.
  • Jessica Drury, Clinical Research Assistant/Regulatory Specialist: Jessica is a graphic and interior designer by training, but she brings her eye for detail and design to RDR as a research assistant and regulatory specialist. Already helping us revamp our email signatures, she'll also be using her talents to help us step our social media game with some more eye-catching graphics and content.
  • Allen W. Graham, Clinical Site Manager: Allen comes to RDR with years of experience as a clinical research coordinator, trial supervisor, and laboratory manager. She brings many great ideas for maximizing clinic efficiency and trial organization. Thanks, Allen, for all your hard work so far!
  • Erin Kistenberg, Undergraduate Intern: Erin is currently a fourth-year student majoring in biology at the Georgia Institute of Technology. She hopes to pursue a career as a genetic counselor and especially enjoys interacting with patients at RDR. Erin has been assisting with the Fibrogen studies, and we are so grateful for all of her help.


This year, we have added many rare indications to the list of clinical trials being conducted by our dedicated team:

  • Prader Willi Syndrome
  • Myotonic Dystrophy Type 1 
  • Spinal Muscular Atrophy
  • Fragile X Syndrome

...and many more are in the pipeline!

All in a rare day's work

“Learning Curve”: Since the spring of this year, we have a new study that requires all hands on deck. We often chat with the team via Google Hangouts. Dillon, one of our newest members, got a message that he needs to be on site at 2am the following day to assist with the study. Without hesitation, he texted back, “I’ll be there!” Little did he know that RDR has a bunch of pranksters who are always looking for a gullible soul. Thankfully, someone remembered to “rescue” Dillon and let him have a decent night of sleep! Phew…


Let’s learn together about the regulations that enable us to be a part of many studies that can and will change the lives of patients living with rare diseases. 

What is the Orphan Drug Act? 

This is a law that passed by Congress in 1983 to provide incentives for the development of medication for a rare disease or condition that affects less than 200,000 people in the United States. This act allows for the following:

  • Tax credits for qualified clinical trials for human testing 
  • Waiver of approximately $3 million in new prescription drug user fee
  • Exclusive marketing for 7 years after drug approval

What is an orphan drug? An orphan drug is a drug for a rare disease or condition. The development of orphan drugs is challenging and complex and, very often, it is disrupted or unable to continue due to lack of funding and financial stimuli.  

What does the FDA do to help? 

  • Facilitates the execution of the Orphan Drug Act through its various offices including the study of biologics, radiology, and medical devices.
  • Provides Rare Pediatric Disease Priority Review Vouchers
  • Offers Orphan Products Grants which help encourage timely development of novel therapies and innovative trial designs with smaller investment while maintaining the safety of trial participants. These grants also allow for many invaluable natural history studies that help “address knowledge gaps, support clinical trials and advance rare disease medical product development.”
  • Organizes Orphan Products Development Events,  such as the annual Rare Disease Day and other public meetings which highlight the status of drug and medical device development and help to educate the public.

Contributed by Trang Vo, PharmD (RDR Pharmacist/Regulatory Specialist) from Retrieved August 28, 2021.