Rare Diseases

Member Services Networks and Communities Translational & Precision Medicine Rare Diseases

Rare Diseases (RD) Community

Description & Goals

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.

Community Connection

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

Leadership

Maria Burian, MD, PhD

Community Chair

Amitava Mitra, PhD

Community Vice Chair

Islam Younis, 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