About the International Rare Diseases Research Consortium (IRDiRC): IRDiRC was formed to unite public and private sector funders of research, patient advocacy groups, and scientific researchers to
advance rare diseases research worldwide.
Not exact matches
«Steep funding cuts for the federal health agencies are counterproductive at a time when innovative
research is moving us closer to identifying solutions for
rare diseases, new prevention strategies to protect Americans from deadly and costly conditions,
advances in gene therapy, new technologies for understanding the brain, and treatments that harness the ability of our immune system to fight cancer.»
New products and developments, such as new drugs for cancer, fresh therapies for
rare diseases, progress in medications for HIV / AIDS, and
advances in stem cell
research had the greatest positive impact.
In new
research, scientists reprogrammed skin cells from patients with
rare blood disorders into iPSCs, highlighting the great promise of these cells in
advancing understanding of those challenging
diseases — and eventually in treating them.
UCLA researchers led by Drs. Peiyee Lee and Richard Gatti at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell
Research have used induced pluripotent stem cells (iPSC) to
advance disease - in - a-dish modeling of a
rare genetic disorder, Ataxia Telangiectasia (A-T).
Overall, despite the dramatic therapeutic
advances made elsewhere in the melanoma field, the poor prognosis of patients with mucosal melanoma mandates continued emphasis on laboratory and clinical
research efforts in this
rare subset of
disease.
This tool is one of the main outcomes of RD - Connect, the EU - funded project that has built a community of patients, clinicians, researchers and bioinformaticians in Europe and beyond, and has
advanced collaboration and data sharing to facilitate and accelerate
research for
rare diseases.
Collaborative Group of the Americas on Inherited Colorectal Cancer aims to
advance research on several
rare forms of inherited colorectal cancers, as well as improve clinical care for patients and families affected by these
diseases.
If used broadly, the recognized platforms / tools / standards / guidelines
advance research and clinical services for
rare diseases.
Scientific
advances can also take a long time, as it is difficult to find sufficient numbers of people with the same
rare disease to enable successful
research.
In the last five years, major
advances in genomic technology, especially in sequencing whole genomes and whole exomes (the portion of the genome that codes for proteins), have given
rare disease research an important boost.
The ultimate goal is to form a national resource to diagnose both
rare and new
disease,
advance laboratory and clinical
research, enhance collaboration and coordination among researchers, and share resulting data and approaches throughout the scientific community.
1:45 PM Osteogenesis Imperfecta: How an advocacy group initiative established collaborative
research for a rare disease, advanced knowledge and care, and fostered an NIH Rare Disease Clinical Research Network to investigate brittle bone d
research for a
rare disease, advanced knowledge and care, and fostered an NIH Rare Disease Clinical Research Network to investigate brittle bone disea
rare disease, advanced knowledge and care, and fostered an NIH Rare Disease Clinical Research Network to investigate brittle bone di
disease,
advanced knowledge and care, and fostered an NIH
Rare Disease Clinical Research Network to investigate brittle bone disea
Rare Disease Clinical Research Network to investigate brittle bone di
Disease Clinical
Research Network to investigate brittle bone d
Research Network to investigate brittle bone
diseases.