Rare Disease Spotlight
Spotlight Newsletter | Rare Diseases Clinical Research Network
Subscribe to the RDCRN Spotlight Newsletter to catch up on the latest rare disease news, features, and insights about research.
Spotlight on rare diseases - The Lancet Diabetes & Endocrinology
As there is no approved treatment for 95% of rare diseases, a diagnosis can be a crushing reality check for patients and their families, rather than bringing ...
Rare Disease Spotlight - Muscular Dystrophy Association
Myasthenia gravis (MG) is an autoimmune disease that leads to muscle weakness. It affects about 14 to 20 out of every 100,000 people. The age of onset varies ...
Rare Disease Spotlight: Hemophilia Treatment Advances
Those with hemophilia B, however, might not begin exhibiting symptoms until later in life. Development in the rare disease drug landscape is expected to ...
About half of these rare diseases are neurological disorders. As we at icometrix strive to improve the lives of people with brain disorders, it is clear-cut to ...
Rare Disease Day and the Spotlight on NORD Member Organizations
Rare Disease States and NORD Member Organization Highlights · Definition: Most frequent cause of severe, persistent hypoglycemia in newborn ...
Spotlight on: Rare diseases - Karolinska Institutet
Rare diseases are diseases that affect fewer than five in 10000 people. But there are many who are affected - in total there are about as many patients with ...
Raising the voice for rare diseases: under the spotlight for equity
People living with rare diseases are calling for equity, to be recognised by the world, and to live their lives like everyone else.
Rare Disease Spotlight: MPS & DMD — a BioCentury podcast
A biomarker breakthrough could shave years off the development of therapies for a group of devastating, progressive ultrarare diseases.
New Consortium Spotlight Articles Offer Look Inside Rare Diseases ...
We are launching a new article series that offers a window into rare diseases research with this issue of the Spotlight newsletter.
Spotlight Editions - Rare Revolution Magazine
Subscribe Now · Rare Disease Day 2024 · CSL Behring Spotlight · Rare and chronic autoimmune diseases · Newborn screening · Hereditary Angioedema · CSL Behring ...
Rare Disease Day and the Spotlight on NORD Member Organizations
NORD's sponsors, media partners, and supporters enable the organization to advance programs to achieve health equity for rare disease families on Rare Disease ...
Advancing rare disease research with powerful protein profiling
With more than 300 million people living with a rare disease worldwide,1 helping to conquer the unique challenges presented by rare diseases is a passion shared ...
Tracing the rise of orphan drug designations over almost 40 years
Rare Disease Spotlight – Tracing the rise of orphan drug designations over almost 40 years ... In the first edition of Pharmaceutical Technology's ...
Spotlight on rare diseases - PubMed
Author · PMID: 30683214 · DOI: 10.1016/S2213-8587(19)30006-3. No abstract available. Publication ...
Spotlight on Rare Disease: Why a patient-centric approach is vital
In 2019, 17 percent of individuals living with rare diseases permanently relocated their home or considered doing so to access proper medical ...
Shining a Spotlight on Rare Diseases - PM360
So, we are putting a spotlight on rare diseases with our first-ever Rare Disease Forum. ... No matter how small the potential patient pool may be, ...
Spotlight on Rare Disease Caregivers - Ultragenyx Patient Advocacy
Forty percent (40%) of rare disease caregivers report having fair or poor emotional or mental health and thirty percent (30%) report having fair or poor ...
Putting a spotlight on Skeletal Rare Diseases
A significant proportion of rare diseases specifically involves the skeletal system, with more than 430 genetic disorders of the skeleton ...
£142 million, 640 projects, 67 (27) charities, 29%: Awards from current AMRC members active in 2018. Inclusion criteria: award was either funded ...