The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME…
According to a story from BioPortfolio, the drug companies bluebird bio, Inc. and the Bristol-Myers Squibb Company have recently announced positive final results for their phase 2 clinical trial. This…
According to a story from globenewswire.com, the biopharmaceutical company Lexicon Pharmaceuticals has recently announced that it has completed its safety review of the initial cohort for its phase 2 clinical…
Personalized Medicine You may have heard of personalized medicine. It's a new wave of healthcare that aims to provide individualized treatment to patients based on their own genetic makeup. The…
According to a story from Medscape, an advisory panel from the US Food and Drug Administration (FDA) has recommended the approval of the drug teprotumumab for intravenous administration as a…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. has announced recently that its phase 3 clinical trial has officially completed enrollment. This trial is testing the…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…
According to a story from prnewswire.com, the biopharmaceutical company InMed Pharmaceuticals, Inc. has recently announced that that company's Clinical Trial Application has been approved in the Netherlands. This approval will…
According to a story from BioPortfolio, the drug developer Rocket Pharmaceuticals recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This study is…
According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…
According to a story from Benzinga, the drug company Abeona Therapeutics, Inc. has recently announced plans to move forward with its phase 3 clinical trial following the lifting of the…
According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The…
Researchers from Brigham and Women's Hospital recently announced a new discovery which could potentially lead to a new multiple sclerosis (MS) therapy. This research has been published in Cell Host & microRNA.…
Therapix Biosciences specializes in cannabinoid pharmaceuticals. Currently, they are working on developing a therapy called tetrahydrocannabinol or THX-110 as a treatment for Tourette syndrome (TS). Additionally, it is being developed…
According to a story from The People's Pharmacy, the US Food and Drug Administration (FDA) has given the green light for the use of generic drugs manufactured in India despite…
According to a story from the Washington Post, rare diseases are a much more widely spread problem than you might think. There are at least 7,000 diseases that are considered…
Jojo, a ten-year-old girl, has become the first person to ever receive a new gene therapy for the treatment of GM1 gangliosidosis. The treatment, called AXO-AAV-GM1, was used in…
Some scientists never have the opportunity to meet a single patient in the community of the disease they work their entire lives trying to understand. This can make them feel…
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
The University of Massachusetts Medical School has recently announced preliminary results from their trial for Tay-Sachs disease. This trial is investigating a gene therapy and researchers are excited about it's…
Pulmonary Hypertension Association The Pulmonary Hypertension Association (PHA) is headquartered in Silver Spring, Maryland. It was formed in 1991 by 4 women passionate about finding a cure. Additionally, they hope…
The medical field is constantly evolving, and rightly so. We should never be complacent with things as they are when it comes to healthcare. There is always more to be…
According to a story from BioPortfolio, Acceleron Pharma and the Bristol-Myers Squibb Company have announced recently that the US Food and Drug Administration (FDA) will review the supplemental Biologics License…
According to a story from Business Wire, the biopharmaceutical company Bristol-Myers Squibb has recently announced that the US Food and Drug Administration (FDA) has granted the company's drug abatacept (marketed…
Surufatinib was recently granted Orphan Drug designation by the FDA. This drug was made for the treatment of pancreatic neuroendocrine tumors (PanNETs). It was created by Hutchison China MediTech…
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