According to a story from Parkinson's News Today, both doctors and patients may delay treatment of Parkinson's disease because of concerns about side effects. The most common treatment for the…
According to a story from BioPortfolio, the biopharmaceutical company AVEO Pharmaceuticals and the lung cancer diagnostic company Biodesix recently released the results from the Phase Ib clinical trial testing a…
The FDA has just announced that they've approved Mayzent for the treatment of relapsing multiple sclerosis (MS). Multiple Sclerosis Multiple sclerosis is a neurological autoimmune disease. It causes a wide…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc. recently announced that the US Food and Drug Administration (FDA) has approved the company's application to…
According to a story from Market Screener, the biopharmaceutical company Imbrium Therapeutics LP recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation for the…
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The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the FDA based on interim results of the clinical trial ADMIRAL, according to an article in EurekAlert for Science News. A…
According to a story from Euroinvestor, the drug development company Resverlogix Corp. recently announced that it has received the necessary $2.9 million in funding for a Phase 2 clinical trial…
According to a story from andreeger.com, gene therapy, a type of medical treatment in which genes are modified in order to treat disease, has become a hot button topic in…
According to a story from businesswire.com, the biopharmaceutical company PellePharm, Inc. recently announced that it has dosed the first patient in the company's Phase 3 clinical trial. This trial is…
According to a story from EurekAlert!, a recent research study has found that a two part immunotherapy combination treatment has the potential to be a useful treatment for high-grade neuroendocrine…
According to a story from Science & Technology Research News, at team of researchers led by Kenneth S. Kosik, who is affiliated with UC Santa Barbara, have made a discovery…
Recently Dr. Janet Maynard, who currently is the director of the US Food and Drug Administration's (FDA) Office of Orphan Drugs Development, wrote an article in which she discusses the…
Happy April! We hope everyone's month is going well! This week, we're highlighting two patient stories and two research stories. First, we have the story of Carolyn, a pemphigus patient…
According to a story from Parkinson's News Today, a recent study found that treatment with an experimental gene therapy called AX0-Lenti-PD was able to improve symptoms for two patients with…
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OncLive recently published the views of several speakers at its State of the Science Summit on breast cancer. Stephanie LaBomascus, MD gave a presentation that highlighted triple negative breast…
According to a publication from World Pharma Today, the 16th annual BioAsia conference saw the greatest ever amount of participation at what is consistently Asia’s largest biotechnology and life science…
At the 2019 annual meeting of The Endocrine Society, ENDO 2019, results from a recent study evaluating burosumab for the treatment of X-linked hypophosphatemia (XLH) were presented. This drug was…
According to a story from BioSpace, the genomic medicine company Sangamo Therapeutics, Inc. and Pfizer, Inc. recently announced the release of interim data from a Phase I/II clinical trial. This…
The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the USFDA based on interim results of the clinical trial ADMIRAL according to an article in EurekAlert for Science News. A total of 371 adult patients…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. When: Saturday, April 27, 2019 Where: West Penn Hospital 4800 Friendship Avenue Pittsburgh, PA 15224 Wintergarden Conference Center – First…
Nonalcoholic fatty liver disease (NAFLD) is a condition in which the buildup of fat in the liver poses health concerns. Nonalcoholic Steatohepatitis (NASH) is a more severe, and more rare…
If you haven’t heard of bullous pemphigoid, you’re not alone, the condition isn’t typically well known even by physicians. For those living with it, this tends to mean a long,…
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A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
Cardiff University in Wales has just announced the launch of their new Medicines Discovery Institute. It’s goal? To help accelerate the development of novel therapies for those living with conditions…
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