The RDCRN social media presence aims to increase awareness of rare diseases, as well as assist with protocol accrual and registrations for the RDCRN Contact Registry.
The Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium aims to advance therapeutic development through study of the relationship between clinical phenotype and underlying genotype, and also through the discovery and development of biomarkers.
Actively engaging patients in research remains an important priority for NCATS. To that effect, we will look at ways to expand opportunities for patient participation in the conception, design, oversight and implementation of research.
TS Alliance serves as a two-way communication conduit representing the needs and priorities of individuals impacted by tuberous sclerosis complex (TSC) to the DSC and spreading awareness of research opportunities to the TSC community.
The Certificate program is a 12 month series of in-person and webinar-based opportunities allowing for synchronous and asynchronous learning opportunities to better understand the practical skills required to succeed in rare disease clinical research.
The Autonomic Rare Disorders Clinical Research Consortium (known also as the Autonomic Disorders Consortium, ADC) investigators and associated patient advocacy groups (PAGs) joined forces in 2008 to establish a broad-based initiative to find better ways to identify rare autonomic diseases, elucidate fundamental mechanisms of their pathogenesis, and discover therapeutic strategies to treatment.
The Genetic Disorders of Mucociliary Clearance Consortium (GDMCC) is comprised of 11 geographically-dispersed sites in North America and associated patient advocacy groups (PAGs). This Consortium studies a variety of rare airways diseases, including Primary Ciliary Dyskinesia (PCD), variant Cystic Fibrosis (CF), airway infection with non-tuberculous mycobacteria (NTM), rare immune disorders, and idiopathic bronchiectasis.
When my daughter was diagnosed with primary ciliary dyskinesia (PCD) in 1991, after seven long years of looking for answers for her chronic lung infections, my first question was 'who is doing research on this disorder?'...
Anyone who has been involved in rare diseases in the past quarter century will have had many opportunities to appreciate the enormous and unique impact of Dr. Stephen Groft's career in the field of rare diseases research. He has been a dominant figure and yet an easily approachable, collaborative and helpful champion of rare diseases research.
