According to a story from SFGate, many survivors of childhood cancers are plagued with financial burdens for the rest of their lives. This is because treatment of childhood cancer can…
The US FDA has awarded Orphan Drug status to the investigational drug NSI-189 for use by people with Angelman syndrome. You can read the source article here, at Nasdaq. About…
According to a story from The Guardian, a recent study suggests that cannabidiol, or CBD, could magnify the effectiveness of treatment for pancreatic cancer. CBD is naturally found in the…
The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…
According to MedPage Today, a phase III study showed that 6 additional months of chemotherapy for maintenance led to a sizable increase in the 5-year overall survival rate for…
According to News Medical, a team of researchers from London have created new technologies that may aid in further understanding Duchenne muscular dystrophy (DMD). Not only this, but the new…
According to BioSpace, a new treatment for pemphigus vulgaris (PV) has been recently approved by the U.S. Food and Drug Administration. The treatment, Rituxan (rituximab), is geared towards adults who have…
The European Medicines Agency has awarded Orphan Drug designation to selumetinib for the treatment of neurofibromatosis type 1. You can read the source press release here, on Merck’s website. About…
According to a story from Hospital Healthcare Europe, The European Medicines Agency approved Kineret last year, an anti-inflammatory drug developed by Sobi. In the UK, Kineret is an approved treatment…
According to a story from Kaiser Health News, many patients that are dealing with chronic pain feel stuck in the debate over opioids in the US. As the ongoing crisis…
According to a story from World Lifestyle, Emmy and Jake were high school sweethearts, destined for love. Indeed, even after years apart, the two found themselves together again after graduating…
According to a story reported by Global News Canada, Michelle Quinlan said it was seven years before doctors correctly diagnosed her daughter, Avery, with Coffin-Siris syndrome. A new project by…
UPDATE: Patisiran has been approved! Check out the story here. According to a story from Xconomy, the U.S. Food and Drug Administration could be close to approving a new drug…
Ehlers-Danlos syndromes (EDS) are a group of rare conditions that affect the connective tissue and can be inherited. Connective tissue lies between tissues and organs throughout the body and helps…
At the 2018 Aplastic Anemia and MDS International Foundation’s (AAMDSIF) International Bone Marrow Failure Disease Scientific Symposium of March 2018, Dr. David Margolis from the Medical College of Wisconsin, and…
In an article by the Fibrous Dysplasia Foundation, the brief and wondrous life of Mauricio Saravia is remembered and celebrated on the advent of his mother’s memoir publication. Mauricio,…
According to a source article on Business Wire that can be found here, the Committee for Orphan Medicinal Products (part of the European Medicines Agency) has recommended that the experimental…
According to a story from PR Newswire, the specialty drug development company Mallinckrodt recently announced that enrollment for trials of its experimental medication MNK-1411 has started. The drug will be…
According to a report by EuroInvestor, biopharmaceutical company Novelion Therapeutics, Inc. was recently granted permission to market their drug MYALEPTA. Novelion intends MYALEPTA to function as a method of treating…
According to a story from Market Screener, the biopharmaceutical company MediciNova, Inc., recently announced its intention to begin a Phase 2/3 clinical trial for its product MN-166. MN-166, also known…
Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease. With fetal gene…
According to a story from businesswire.com, Otsuka Pharmaceutical Europe Ltd recently announced that its product JINARC, also known as tolvaptan, now has an expanded therapeutic indication after a recent approval…
Patient Worthy exists because there's just not enough visibility for the nearly 30 million Americans who have been diagnosed with disorders that are rare or have limited treatment options. So…
According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the…
Diseases affecting the brain are often pose numerous difficulties in research. One such challenge is that animal models of the diseases never quite replicate the human reality. In mice, for…
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