Rare Diseases (RD) Community

The Rare Diseases (RD) Community is a home for multidisciplinary researchers who are interested in improving the efficiency of research on rare diseases to enhance patient outcomes. It serves as a place to develop scientific programs and disseminate impactful publications, presentations, and other resources that lead to rich discussions among members to help develop and improve best practices for drug discovery, development, regulation, and utilization to treat rare diseases.

Past Webinars:
The webinars below can be accessed in the Members Only Webinar Library: Access Library
  • The Application of Model-Informed Drug Development in Rare Disease Development
  • From Lab to Clinic: Model-based Approaches in Rare Disease Drug Development
  • The Application of Clinical Pharmacology for the Treatment of Rare Diseases
  • Cell and Gene Therapy: Translational Medicine and Real-World Data
  • Development of Gene and Cell Therapies for Rare Diseases: Challenges, Opportunities and Regulatory Interactions
  • Patient/Family-Driven Gene Therapy Development for Rare Diseases

I Younis

Islam Younis, PhD

Community Chair


M Burian

Maria Burian, MD, PhD

Community Vice Chair


M Ahmed

Mariam Ahmed, PhD

Community Past Chair


Steering Committee
Piet Van Der Graaf, PharmD, PhD
Noha Rayad, PhD
Gina Patel, PhD
Kefeng Sun,PhD
Amitava Mitra, PhD
James Cloyd, PharmD
Salwa Albusaysi, PharmD, PhD
Yousef Roman, PharmD, PhD
Mujal Patel, PhD
Rajesh Krishna, PhD
Reena Kartha, PhD
Indranil (Neil) Bhattacharya, PhD
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