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 ...
STAT2d
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 ...
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 ...
Psychology Today4d
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 ...
STAT3d
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 ...
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 ...
GEN3d
Rare Bleeding Disorder, HHT, Treated Successfully with Cancer Drug in Clinical Trial
An NIH-supported clinical trial of pomalidomide in treating hereditary hemorrhagic telangiectasia stopped early due to ...
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 ...
Kerala Issues Alert As Amoebic Meningoencephalitis Cases Spark Health Emergency
There are very few scientific studies and study results about this rare disease. Statistics indicate that only 2.6 out of 10 ...
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 ...
Morningstar1mon
Ambry Genetics Announces New Multiomic Exome Test for Improved Rare Disease Detection
ExomeReveal™ test expands Ambry Genetics’ clinical exome capabilities and applies its RNA analysis expertise to rare disease testing Ambry Genetics, a leader in clinical genomic testing and a ...