Dr. Rothenberg, the Principal Investigator for CEGIR, has been awarded the highest honor bestowed by the International Eosinophil Society for his major contributions to the advancement of our understanding of the eosinophil.
NEPTUNE has embraced the concept of Precision Medicine proposed by the Institute of Medicine in 2011. The Precision Medicine strategy proposes the development of new disease definitions generated by a multilayered analysis of the disease course while patients receive their usual clinical care in observational studies.
Through a recently awarded Clinical Trial Readiness grant from NINDS, CReATe investigators are now tapping into the invaluable resource that is the CReATe Biorepository in an effort to clinically validate leading biological fluid-based biomarker candidates to aid the design and implementation of future clinical trials.
Through research, the BBDC is discovering new biomarkers of the disease that will lead to a deeper understanding of underlying pathophysiology of OI as well as assessing response to therapies
The Porphyrias Consortium (PC) seeks to continually improve the diagnosis and treatment of the eight major porphyrias.
Investigators from the 'MILES' (Multicenter International LAM Efficacy of Sirolimus) trial recount the unique path it took seeking FDA review of their drug.
The American Partnership for Eosinophilic Disorders (APFED) is one of several Patient Advocacy Groups collaborating with the Consortium of Eosinophilic Gastrointestinal Disease Researchers to conduct research. APFED provides support in many ways, such as engaging the patient community, participating in committee work, and even pledging supplement funding to support future research.
An article published in Orphanet Journal of Rare Diseases shows the integral partnership between RDCRN investigators and the associated patient advocacy group members, as well as how this partnership has enhanced recruitment for protocols, study design, and consortium training programs.
The Rare Diseases Clinical Research Network (RDCRN) sponsored the 4th Conference on Clinical Research for Rare Diseases (CCRRD) on Thursday, November 3, 2016 in Washington, D.C.