According to a story from pm360online.com, the U.S. Food and Drug Administration (FDA), has given Priority Review status to eltrombopag (marketed as Promacta) after impressive clinical trial results. Eltrombopag was…
According to a story from Telegraph & Argus, 56 year old Andrew Johnson is getting an innovative new procedure that is offered on the UK's NHS in order to relieve…
According to a story from news-medical.net, Mariona Graupera and Frances Viñals, along with Dr. Antoni Riera-Mestre, announced the completion of research that could lead to a new potential treatment for…
According to a story from news-medical.net, the results of a recent research trial demonstrate that the radiation therapy drug AZEDRA (ultratrace iobenguane I131) is an effective and safe treatment option…
May 15 to June 15 is designated as Tourette Syndrome Awareness Month, a rare neurological disorder that causes involuntary movements or vocalizations known as tics. Examples of these tics include repetitive blinking, clearing…
Spinal muscular atrophy is a genetic disease with life threatening implications. In order to create effective therapies, doctors need the ability to target the root genetic mutation and stop it.…
A Phase II trial of the experimental treatment SurVaxM, under development by the biotechnology company MimiVax LLC, has produced encouraging interim results for treating patients with newly diagnosed glioblastoma. The…
The Boston-based biopharmaceutical company Berg LLC has announced that their experimental drug candidate BPM 31510 (ubidecarenone) has been awarded Orphan Drug designation by the US Food and Drug Administration (FDA)…
The United States Food and Drug Administration (FDA) has granted the drug tafamidis Breakthrough Therapy designation for treating patients who have transthyretin cardiomyopathy. The full article can be read here,…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the Phase 2 trial for its proprietary, investigational product sotatercept. The medication is in development…
The PBCers Biannual Conference recently took place in Houston, Texas. The 2018 PBCers Conference was highly informative with topics ranging from the basics of Primary Biliary Cholangitis, dietary tips, updates…
The United States Food and Drug Administration (FDA) has approved the label update for the medicine Taltz (ixekizumab) 80 mg/mL injection for psoriasis that affects the genital area. You can…
Fresh off of Memorial Day, there is no better documentary to watch right now than HBO's John McCain: For Whom the Bell Tolls. The Arizona senator announced last July that he…
Two organisations, NeuroVive Pharmaceutical AB from Sweden and Yungjin Pharm Corporation based in South Korea, have announced encouraging initial results from a Phase I clinical trial of the experimental medicine…
The companies Centogene and Orphazyme are collaborating on a clinical trial of the experimental small molecule therapy arimoclomol for Gaucher disease. The full article can be read here, at BusinessWire. Gaucher…
A drug called Hemlibra that is currently only approved for treating a subset of patients with haemophilia A has been found to be effective for wider use, reports Hemophilia News…
When others learn that you are the parent of a child with a rare disease, they most likely correctly assume that doctor appointments and hospitals might be involved. Yet few…
According to a story from CNN, substitute teacher Cynthia Tisdale was widely admired by her family and friends. Cynthia was murdered in the Santa Fe High School shooting on May…
Military veterans are known to have twice the risk of developing amyotrophic lateral sclerosis (ALS) compared to the general population, but researchers are yet to understand why. The full story can…
According to a story from kait8.com, the company Harmony Biosciences, Inc., has recently announced that the U.S. FDA has given Fast Track and Breakthrough Therapy designations for its experimental therapy…
According to a story from apnews.com, sickle cell disease gets very little research or funding despite that fact that about 100,000 people in the U.S. are affected by it. The…
According to a story from Medical Xpress, chronic fatigue syndrome is one of the most poorly understood diseases. Although the name chronic fatigue syndrome seems to merely suggest that patients…
As of last Monday, the United States Food and Drug Administration (US FDA) its approval of a new drug to treat thrombocytopenia in cases of chronic liver disease (CLD). Keep…
According to a story from Worldpharmanews, a PARP inhibitor drug that is commonly used to treat ovarian cancer for patients with BRCA gene mutations could also be effective against pancreatic…
In order to keep up with the quick-growing field of gene therapies, the United States Food and Drug Administration (FDA) announced it will provide faster methods of approval for new…
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