Research paper boosts understanding of rare disease.

We provide a united voice for the rare disease community by capturing the experiences of patients and families. We work with our supporters to raise the profile of rare diseases across the UK. We seek to bring about lasting change offering better health and quality of life for individuals and families affected by rare diseases.

NORD’s Director of Research Programs Publishes Article on Rare Disease Research in the 2020 Rare Neurological Disease Special Report “Now Is The Time For Research On Rare Diseases details the importance of natural history studies, the role of patients and caregivers as research partners, and the power of collaboration.

The journal will feature independent, original research articles as well as in-depth review of specific rare diseases and therapeutics, and opinion pieces from regulators, patient advocates and leading industry and academic researchers focused on rare diseases.

Established by Congress under the Rare Diseases Act in 2002, RDCRN is an initiative of the Office of Rare Diseases Research (ORDR) at the National Institutes of Health’s (NIH’s) National Center for Advancing Translational Sciences (NCATS). View our network of 23 active consortia or research groups.

Caring for Your Patient with a Rare Disease Patients with a rare disease present unique challenges for primary care providers (PCPs). Obstacles to caring for these patients include diagnostic delays and a lack of information, expertise, and treatment options for many rare diseases.

For Rare Disease Day, Professors Uta Griesenbach and Eric Alton tell us why rare diseases are the hidden priority of scientific research. A rare disease, also known as an orphan disease, affects by definition less than five in 10,000 (or 0.05%) of the general population.

Rare diseases are at the forefront of patient engagement in medical research and drug development. They are fundamentally changing the way that new treatments are delivered, creating a focus on patient need and patient experience. Rare diseases are some of the conditions targeted by the first pioneering gene therapies to reach patients.

Registries can be an incredibly useful tool in gathering data on patient usual care, current treatment landscape(s), and long-term clinical outcomes, as well as other uses noted by recent FDA guidance, 2 to better understand the impact to the disease population and strategically plan for additional real-world patient research and treatment development.

Centre for Rare Disease Studies (CRDS) Birmingham We explore, promote and contribute to the latest developments in rare disease research, with the goal of improving the lives of the many people living with rare diseases.

The Rare Diseases Clinical Research Network is an initiative of the Office of Rare Diseases Research. National Center for Advancing Translational Sciences (NCATS) funded through a collaboration between NCATS and the National Institutes of Health.

Rare Disease Clinical Research Untapped potential in MENA March 2019 A White Paper by Clinart MENA in collaboration with LongTaal Author: Rachel V. Brady Access live data. 2 Rare Disease Landscape Around the turn of the century, the tidal wave of success from numerous.

Pfizer is adopting an innovative and collaborative approach to the development of new medicines for patients with rare diseases. We have a track record of creating innovative strategic partnerships with academic institutions, patient advocacy groups, and commercial enterprises to accelerate the development of novel therapeutics across the entire spectrum of rare diseases.