According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…
EPP Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come…
According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…
Supplies of a Critical Bladder Cancer Drug are Dwindling Bob Field, a 72-year-old New York banking executive, received a call from his urologist’s office canceling his next appointment. According to…
According to a story from ncbiotech.org, a new partnership between Vertex Pharmaceuticals and RNA-based therapeutics company Ribometrix aims to develop potential treatments for serious diseases, including Huntington's disease, which is…
According to a story from PR Newswire, the biopharmaceutical company Clene Nanomedicine, Inc., has issued an announcement in regards to its Australian subsidiary. This subsidiary has been awarded a grant…
According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…
In a recent press release, Massachusetts-based biotech company Amylyx Pharmaceuticals announced the completion of dosing in the 24 week study of their experimental amyotrophic lateral sclerosis treatment AMX0035. About Amyotrophic…
A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole. About the…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
Geisinger Health System is based out of Pennsylvania. They have recently initiated a novel program that has goals to make DNA sequencing a standard part of routine clinical care. Next…
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…
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