According to a story from news-medical.net, a team of researchers have developed a potential new treatment for polycystic kidney disease (PKD), a rare disorder that ultimately causes organ failure. The…
According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…
NASH Nonalcoholic Steatohepatitis (NASH) is a rare disease in which hepatic inflammation and injury to the cells occur as a result of steatosis (hepatic fat accumulation). Severe cases of NASH…
According to a story from Biotech 365, the immuno-oncology company Mateon Therapeutics Inc. has recently announced that the company's investigational product candidate OT101 has earned the US Food and Drug…
Prevalence of MS in Korea In South Korea, rates of multiple sclerosis (MS) are generally low. This is because the disease is most prevalent in areas where there is lower sun…
According to a story from The Sunday Post, it is an interesting time to be a cystic fibrosis patient in the UK these days. Just weeks after The Scottish Medicines…
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Waukesha Wisconsin Police Captain Dan Baumann recently held a news conference that was described in an article published in Scientific American. Captain Baumann said that parents of a teenage boy showed…
A recent publication from the US Food and Drug Administration announced the regulatory authority's approval of Nourianz (generic name istradefylline) for Parkinson's patients during "off" episodes. "Off" episodes are windows…
The Center for Chronic Illness is a nonprofit group that is based out of Seattle, Washington. The goals of this organization include the provision of support and educational resources to…
According to a story from Yahoo! News, Dorothy Dunnington is beginning to wonder if she will ever fully understand the circumstances surrounding the death of her sister Chrissy, who was…
According to a story from The Guardian, Jeremy Corbyn, the leader of the UK Labour Party, announced a remarkable plan at the most recent Labour party conference that has the…
The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
According to a story from Financial Buzz, the biotherapeutics company Bellerophon Therapeutics Inc. has recently announced that it has earned Orphan Drug designation from the US Food and Drug Administration…
According to a story from Fabry Disease News, the first patient in a clinical trial testing the investigational gene therapy FLT190 has reportedly been dosed. FLT190 is being developed as…
Cystinosis Cystinosis is a rare disease that causes cystine, an amino acid, to build up in the body's organs. However, there is treatment that can prevent this accumulation. Normally, patients…
On May 29th, 2019, the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) alongside other members of the fibrodysplasia ossificans progressiva community, sat down for an hour long 'listening session' with representatives…
Most young people are familiar with vaping: nicotine or THC repackaged in a sleek and less stigmatized device. It is less obviously recognizable as destructive as cigarettes in part because…
According to a story from outsourcing-pharma.com, the company Firma Clinical Research, which provides health care services to patients in clinical trials, has been getting a lot of business lately. This…
According to a story from sectorpublishingintelligence.co.uk, the drug company INVENTIVA's lead product candidate, known as lanifibranor, has earned Fast Track Designation from the US Food and Drug Administration (FDA) as…
According to a story from finanzen.ch, the US Food and Drug Administration (FDA) recently approved the drug rituximab (marketed as Rituxan) as a treatment in child patients aged at least…
The National Organization for Rare Disorders (NORD) is hosting its 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit on October 21-22 in Washington, D.C. NORD is a great organization and…
According to a story from American Nurse Today, the US Food and Drug Administration (FDA) approved a treatment on September 6th, 2019 for interstitial lung disease associated with scleroderma, a…
According to a story from Angioedema News, the UK's National Institute for Health and Clinical Excellence (NICE) has officially recommended the drug lanadelumab (marketed as Takhzyro) for coverage under the…
According to a story from BioSpace, the biotechnology company BioMarin Pharmaceutical Inc. has recently announced its submission of a clinical trial application with the UK's Medicines and Healthcare Products Regulatory…
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