According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…
According to a review in the academic journal Clinical Epigenetics, epigenetic therapy could be a potential future treatment option for ovarian cancer. Epigenetics refers to genetic changes, sometimes heritable, that take…
According to a story from BioPortfolio, the biotechnology company Saniona has released an update in regards to the company's Phase 2a clinical trial of Tesomet, which is being tested as…
Rare disease isn't as rare as it sounds. Ten percent of Americans have a rare disease-- and there are almost 7,000 known rare diseases. A rare disease, also known as…
According to a story from Fierce Biotech, the results of a year-long study examining the utility of the groundbreaking gene-editing technology CRISPR in treating the rare genetic disorder Duchenne muscular…
According to a story from Hemophilia News Today, the drug developer Spark Therapeutics, Inc., has recently released several updates in regards to a number of its experimental gene therapies that…
Patient Worthy has the distinct pleasure of attending the Sixth Annual Rare Disease Genomics Symposium at the University of Alabama at Birmingham (UAB) this weekend. Today, we heard from lead…
Massimo Federico, MD, director of the medical oncology unit at the University of Modena and Reggio Emilia, Italy, was quoted in Cancer Network as saying that “Achieving a complete clinical and…
ADA-SCID ADA-SCID is a form of Severe Combined Immunodeficiency (SCID) which is caused by Adenosine Deaminase Deficiency. A lack of the ADA gene in the body results in impaired development of the…
According to a story from BioSpace, the biopharmaceutical company DelMar Pharmaceuticals, Inc., has recently announced an update to its Phase 2 clinical trial. This clinical trial is testing the experimental…
The very first patient in Medigene AG's clinical trial examining MDG1011 has officially received the treatment. This is the company's first human clinical trial for this TCR therapy. The trial…
The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…
Happy Rare Disease Week! This week has been all about rare diseases. Today, we're highlighting an article from a PW contributor with FAP, followed by a story describing some shortcomings…
Lupus Nephritis Lupus nephritis (LN) is one of the most common complications of systemic lupus erythematosus (SLE). It affects approximately 100,000 patients in the United States alone. Basically, the patient's…
A recent article in CheckOrphan describes a study that was conducted by Researchers at the Lysosomal and Rare Disorders Research and Treatment Center in Vancouver, B.C. The study examines the…
Proteus Syndrome Proteus syndrome is a rare disease caused by the overgrowth of tissues including the skin, adipose tissue, and parts of the central nervous system. It also affects the…
Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…
This is the story of five-year-old Aubree Fox who has recently been diagnosed with hydrocephalus, stenosis, and an aneurysm in her brain. This slew of life-threatening diagnoses were all a mystery…
According to a story from Gene42, a new rare disease platform called Genomics4RD has been launched in Canada. This is the first national research platform dedicated to rare disease to…
Ofatumumab as a Single Agent Results of ofatumumab administered as a single agent with an extended induction (initial treatment) brought about a high response rate and it was well tolerated among follicular…
NICE stands for the National Institute for Health and Care Excellence in England. This organization is responsible for evaluating the cost effectiveness of all medications as they come to the…
“There Is No Gene for the Human Spirit”—from the movie Gattaca, 1997 Whether it’s distant uncles purported to be horse thieves in the early days of the…
The treatment of metastatic renal cell carcinoma (RCC) has evolved over the last ten years and investigators now look towards combination therapies to improve response rates and durability for…
According to a story from EurekAlert!, a recent study revealed that the surgical removal of the thymus gland, known as a thymectomy, was able to provide long-term benefits for patients…
For years the accepted estimate of multiple sclerosis (MS) patients living in the United States was 400,000. A recent study, funded by the National Multiple Sclerosis Society (NMSS) has confirmed that number is…
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