pharmaphorum2d
Rare disease groups: scaling up from the kitchen table
2015. About Rare Diseases. Online. Accessed 8 April 2015. 2 Global Genes Project. RARE Diseases: Facts and Statistics. Online. Accessed 8 April 2015. 3 Shire. 2013. Rare Disease Impact Report ...
pharmaphorum2d
Meeting the challenges of rare disease treatment adherence
Rare conditions present their own difficulties in terms of medicines adherence, but HCPs, pharma and patients can pool their experience to develop effective, tailored support. It is well ...
Medical Xpress5d
News on rare disease
Genetic variants linked to a rare form of bicuspid aortic valve disease that affects young adults and can lead to dangerous and potentially life-threatening aortic complications have been ...
Commentary: Congress has just weeks to help children with rare diseases
A rare-disease federal incentive program critical to biotech R&D is in jeopardy, writes Leslie Williams, president and CEO of ...
STAT1d
FDA approves first drug for rare neuron disease, Niemann-Pick type C
Parents pushed for years for treatments for the neuron disease Niemann-Pick type C. After setbacks, one drug now has FDA ...
STAT2d
House panel passes pediatric rare disease bill, setting it up for government funding bill
A House committee on Wednesday passed a bill to renew a program aimed at aiding development of drugs for rare diseases in ...
US FDA approves Zevra's treatment for rare genetic disease
The U.S. Food and Drug Administration approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the ...
ThePrint on MSN8d
New hope for rare disease patients: Rs 900-cr govt project to get high-value drugs at lower rates in works
India has 8.4 to 19 crore rare disease patients, according to health ministry estimates. But current beneficiaries in most ...
Psychology Today3d
How I Survived a Rare Autoimmune Disease
I walked out of my marriage and immediately fell ill. I was ill for two years. At first, I thought it was a cold that would ...
Fox News20d
Thanks to Terry Pirovolakis, there's now a treatment for SGP50 — but most families can’t afford it
When his infant son was diagnosed with a rare disease, a Canadian father was dismayed to discover there was no treatment or cure. So he set out to make one himself. Terry Pirovolakis, an IT ...
kffhealthnews3d
House Bill To Extend Pediatric Rare Disease Aid Clears Committee
Passage of the measure would tee it up to be added to government funding legislation. It also would ensure the voucher program would be in place another five years. Meanwhile, the House rejected the ...
Medical Xpress26d
AI-powered, big data research enhances understanding of systemic vasculitis
Researchers from the School of Medicine and the ADAPT Centre at the School of Computer Science and Statistics ... a group of rare and complex autoimmune diseases. The study, part of the FAIRVASC ...