In China, rare diseases affect over 20 million people, with the impact of these conditions highlighted through Rare Disease Day and the story of a young girl in Shenzhen with Tuberous Sclerosis Complex. High treatment costs...
Home Global At Recordati Rare Diseases, we are Rare diseases aren’t just in our name: they are at the core of all that we do. We work side-by-side with rare disease communities to raise awareness, educate, and advocate for improved diagnosis. Together, we can celebrate our successes ...
About us Big thinking for people with rare diseasesEverything we do is driven by the needs of people who have a rare disease:Our team believes that each person with a rare disease has the right to the best treatment. We have nearly 30 years' experience in bringing orphan drugs to market...
Contact Us SolutionsTherapeuticsRare Diseases We rise to the challenge with you Global reach and impact:IQVIA's rare disease solutions span thousands of clinical sites worldwide, ensuring broad access and efficient trials. Your work brings hope to over 350 million people affected by more than 7,00...
The article discusses research study on the pharmaceutical prices of rare disease drugs in the U.S., presented at the 16th Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research.EBSCO_AspPharmacoEconomics & Outcomes News...
RARE Revolution MagazineTM, RARE Youth RevolutionTM and TIDE Patient Engagement are published and created by NRG Collective Ltd, a not-for-profit publishing company specializing in rare disease content. hello@rarerevolutionmagazine.com Privacy Policy ...
Type 1 galactosemia is a rare genetic disease that can be life-threatening in newborns and can lead to lifelong cognitive, neurological, and speech complications, as well as primary ovarian insufficiency in girls and women. 123456789101112 Disease Categories Autoimmune and Auto-inflammatory Disorders ...
1.occurring or found infrequently; markedly uncommon:a rare disease. 2.having the component parts loosely compacted; thin:rare gases. 3.unusually great. 4.admirable; exemplary:She showed rare tact in inviting them. [1350–1400; Middle English < Latinrārusloose, wide apart, thin, infrequent] ...
State-of-the-art in rare disease education: available resources and the needs across education and training continuum There is no health without the healthcare workforce: if we want to respond to unmet needs of PLWRD and their families, we need to fundamentally rethink RD education and HWF dev...
In 2018, the company obtained the commercialisation rights to pegunigalsidase alfa (PRX-102) in the US. PRX-102 is an investigational drug being developed in Phase III clinical trials to treatFabrydisease. Chiesi Global Rare Diseases head Giacomo Chiesi said: “We are dedicated to making rapid...