Poster Descriptions
There is remarkable work being discussed through posters this year. Listed here are descriptions of many of the poster submissions for the NORD Summit. Topics include the latest in innovative research, medical education advancement, patient community building and more.
To view the posters in their entirety, register here for the NORD Summit.
Innovative Research
- Effects of Bacterial Products on PMP Tumor Cells
- Fostamatinib for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) Results of the Phase 2, Open-Label Study
- Fostamatinib, an Oral Spleen Tyrosine Kinase (SYK) Inhibitor, as Second-Line Therapy for Immune Thrombocytopenia (ITP)
- Identifying Causative Variants in Patients with Monogenic Stone Disease
- Integrated Study Results of Teprotumumab The First Drug US FDA Approved for the Treatment of Thyroid Eye Disease
- Next-Generation Phenotyping (NPG) for Cat-Eye Syndrome
- PaVe-GT: Paving the Way for Rare Disease Gene Therapies
- Physical Function Clinical Outcome Assessments for Rare Disorders and Sarcopenia Planning Core Outcome Sets with FDA
- RT001 in Progressive Supranuclear Palsy (PSP) In-Vitro and Clinical Results in 3 Patients
Medical Education Advancement
- A Model of Medical Education That Teaches Doctors to #DareToThinkRare
- Academic and Community Collaboration to Accelerate Kabuki Syndrome Awareness, Research, and Clinical Care
- Access for Patients and Caregivers
- Characteristics of Generalized Pustular Psoriasis (GPP) Patients Results from the Optum® Clinformatics™ Data Mart Database
- Educational Impact of Digital Learning Spinal Muscular Atrophy Education for Clinicians and Caregivers
- Granulomatosis with Polyangiitis A Rare Condition with Otolaryngologic Manifestations
- Patient Advocate-Driven Advancement of Clinical Recommendations in Ultra-Rare Disease The CLN1 Batten Disease Experience
- Update in the Management of Bell’s Palsy
Other
- Accelerating Research Through A Patient-led Collaborative Network
- Pharmacist-identified Gaps in the Expanded Access Process at an Academic Medical Center
- Resilience Education Supporting the Emotional Wellbeing of Those with a Progressive Disease
- Understanding Parent and Expert Concerns and Experiences in Rett Syndrome Qualitative Research to Inform Outcome Measure Development
Patient Community Building in Rare Diseases
- An Update on Treatment Data from the Desmoid Tumor Research Foundation
- Building a Global Rare Disease Coalition Beneficial Outcomes From the Establishment of an International Alliance Community
- GM2DR – The Tay Sachs and Sandhoff Disease Multinational Patient Reported Data Registry
- Managing Online Survey as a Successful Global Rare Disease Patient Community Building Model
- Payer Survey Highlights Confusion About the Definition and Utility of Real World Evidence in Rare Disease
- Sarcoma Coalition We Are Here For You
- Seeing to the Continuity of Healthcare in an era of Physical Distancing