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Retricted access

Research article

First published 2010-1

The National Organization for Rare Disorders (NORD)

Stefanie Putkowski

NASN School Nurse

Retricted access

Other

First published 2000-7

The Patient Advocate Perspective on Preparing for FDA Advisory Committee Presentations

Abbey S. Meyers

Therapeutic Innovation & Regulatory Science

Open access

Research article

First published 2017-12-18

Creating a multi-center rare disease consortium – the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR)

Katherine Cheng, Sandeep K. Gupta, Susanna Kantor [...]

Translational Science of Rare Diseases

Open access

Research article

First published 2019

The initiation,design,and establishment of the Desmoid Tumor Research Foundation Patient Registry and Natural History Study

Kelly A Mercier, Darragh M Walsh

Rare Tumors

Retricted access

Other

First published 2007-08

Spinal Muscular Atrophy: Classification,Diagnosis,Management,Pathogenesis,and Future Research Directions

Felina V. Kostova, Virginia C. Williams, Jill Heemskerk [...]

Journal of Child Neurology

Retricted access

Research article

First published 1992-4

Neurometabolic Diseases at a National Referral Center: Five Years Experience at the King Faisal Specialist Hospital and Research Centre

Pinar T. Ozand, Edward B. Devol, Generoso G. Gascon

Journal of Child Neurology

Open access

Review article

First published 2023

Patient-centered pharmacovigilance: priority actions from the inherited bleeding disorders community

Fiona Robinson, Sonji Wilkes, Nathan Schaefer [...]

Therapeutic Advances in Drug Safety

Retricted access

Research article

First published 1999-6

Managers and Mental Health: Mental Illness and the Workplace

Richard L. Schott

Public Personnel Management

Open access

Review article

First published 2025-01

A Community Effort to Develop Common Data Elements for Pre-Clinical Spinal Cord Injury Research

Britt A. Fedor, Abel Torres-Espin, Romana Vavrek [...]

Neurotrauma Reports

Open access

Research article

First published 2023

Emerging roles and opportunities for rare disease patient advocacy groups

Amy M. Patterson, Megan O’Boyle, Grace E. VanNoy [...]

Therapeutic Advances in Rare Disease

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