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Alzheimer’s disease (AD) is a neurodegenerative disease and the major cause of impaired memory affecting the older population. Reported cases are rapidly increasing. So far, clinical trials have failed…
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The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…
A minimum of seven people have died after contracting Eastern Equine Encephalitis (EEE), a rare disease carried by mosquitoes, bringing number of total people affected to 27 in six states.…
September 25th is International Ataxia Awareness Day! The National Ataxia Foundation (NAF) has great resources to help spread the word and show our solidarity for the Ataxia patient community; on…
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Fierce Biotech recently focused on a report by The BMJ, a British science journal, on the validity of clinical trials which are critical to decision making by patients and…
Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…
According to a recent publication from the Duke University School of Medicine, Dr. Rodger Liddle believes Parkinson's disease may have origins in an unlikely place — the gut. Liddle, a…
According to a story from delmarvanow.com, a nine year old boy named Trey Kearns has progressive familial intrahepatic cholestasis (PFIC) a rare disease that affects the liver. The symptom that…
September 24th is Familial Hypercholesterolemia (FH) Awareness Day! Let's take a closer look at FH, and how we can help make this day productive for the FH -- and rare…
According to a story from scienmag.com, a pair of five year grants that were awarded to Dr. Mark Zylka will go towards projects focused on autism and the rare disease…
According to a story from the Washington Post, Bob Schwartz is a walking, talking medical mystery. He lives with a strange and diverse array of difficult symptoms, and after being…
Outcomes of a pilot study on Gaucher disease using a mobile phone app and wearable technology demonstrate a more accurate approach to disease assessment. Gaucher disease is a lysosomal storage…
According to a story from Benzinga, the medical device company V-Wave Ltd. has recently announced that a product in development by the company has earned Breakthrough Device designation from the…
According to a story from clinicaltrials.gov, the Children's Hospital of Philadelphia is sponsoring a study that has the potential to make mitochondrial disease research and drug development much easier. The…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is a concept a few decades…
According to a press release from the California-based biotechnology company Ardelyx, the US Food and Drug Administration (FDA) recently approved the company's irritable bowel syndrome drug candidate Ibsrela (generic name…
A recent publication from BMC Pediatrics outlines a case study of Kawasaki disease in a group of infants from Italy. Because Kawasaki disease is a rare disease (with only an…
According to a press release from CTD Holdings, the American Food and Drug Administration (FDA) recently approved an Expanded Access application from a physician to treat a pediatric Niemann-Pick disease…
According to a story from ANCA Vasculitis News, a recent study from Spain is highlighting the need for an updated classification system for ANCA-associated vasculitis that can predict patient prognosis…
According to a press release from global pharmaceuticals titan GlaxoSmithKline, the US Food and Drug Administration (FDA) recently expanded approval of the Company's eosinophilic asthma treatment Nucala (generic name mepolizumab)…
There are currently 11 treatment options for multiple sclerosis (MS) in the United States. Some of these are symptomatic treatments, and some actually slow progression of the disease. Before 2009…
A recent study, published in Nature, examined whether or not stem cells could be modified to reduce their chance of rejection. Stem cell therapies are utilized in a wide array…
A recent publication from Specialty Pharmacy Times noted that the rapidly-growing number of available human immunoglobulin treatments for primary immunodeficiency disorders could pose challenges for specialty pharmacists when treating patients.…
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Glen Sauber successfully overcame testicular cancer and was symptom-free-- until three years later when he began to experience neurological symptoms. According to a recent article in the San Francisco Chronicle,…
According to a publication from BioSpace, researchers from Michigan's Memorial Healthcare Institute for Neurosciences and Multiple Sclerosis are taking full advantage of recent technological advances in neurofilament light chain testing…
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