In the past, there have been little to no concrete studies evaluating the relationship between hereditary angioedema (HAE) and cancer risk. However, researchers questioned whether some sort of relationship could…
According to a recent article, a new stem-cell-based gene therapy is showing promising results for treating beta (β)-thalassemia in a phase 1 clinical trial. Beta Thalassemia Beta thalassemia is a…
How does long-term non-steroidal anti-inflammatory drug (NSAID) use influence health? Do NSAIDS used by older adults influence the development of any other conditions or adverse reactions? According to the Nephrology…
Biogen Inc. recently announced data that included extended results from its earlier Phase 3b NOVA study on the administration of natalizumab and the evaluation of the drugs Tysabri® and Vumerity. …
What if researchers could change DNA processes within brain tumors to improve overall survival rates and patient outcomes? According to Medical XPress, researchers found that L-methylfolate, in conjunction with the…
Newborn screening (NBS) is a public health service within the United States through which babies are screened for medical conditions that otherwise wouldn't be apparent at birth. Many of the…
Today, follow along on a case study exploration to see how a 52-year-old woman finally received a diagnosis of stiff person syndrome (SPS), a rare neurological disorder. According to the…
Is there a relationship between increased sun exposure and breast cancer risk? At first glance, you might think, “Sure. Sun exposure probably increases the risk.” However, according to an article…
According to a recent article, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB recently announced the European Commission (EU) has approved Aspaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal…
It’s no secret that stress can be both psychologically and physically damaging. But how does stress influence the way that our bodies respond to certain conditions? According to Medical XPress,…
When she was born, Savannha Aretino was diagnosed with hypophosphatasia, a rare genetic disorder characterized by bone and teeth development abnormalities. Now, twenty-three years later, Savannha is working to not…
According to a recent article, a study shows that treatment with gene therapy reduces the need for patients with non-β0/ β0 genotype Beta-thalassemia (β-thalassemia)to get transfusions. Beta-thalassemia (β-thalassemia) Beta thalassemia…
The Pitt Hopkins Family Conference & Scientific Symposium (PHRF Conference) will be held June 23rd through June 25th, 2022 in Chicago to raise awareness and share research updates for Pitt…
Diagnosing amyloidosis (abnormal protein in tissue) is a complex process. Over eighty percent of amyloidosis patients are undiagnosed. The disease takes hold after the blood plasma cells in the bone…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
The Pulmonary Hypertension Association recently announced it is offering junior investigators who are interested in pulmonary arterial hypertension (PAH) a two-year grant. Pulmonary Arterial Hypertension (PAH) Pulmonary arterial hypertension (PAH)…
PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
It’s January 2022 – have you checked on your thyroid health lately? If not, consider learning more about thyroid disease and what your thyroid does for the body. Why…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
The largest study of benign adrenal tumors has just been completed. Results have been published in the Annals of Internal Medicine. Specifically, this study examined the effect of mild autonomous cortisol…
Saniona has just announced that their Phase 2b clinical trial examining Tesomet as a treatment for Prader-Willi syndrome (PWS) will be beginning soon. This treatment is a combination therapy which…
Nonalcoholic steatohepatitis is a liver disease, causing fat to build up in the liver. The resulting inflammation may lead to liver cancer or cirrhosis (scarring) and possible liver failure. As…
Many people living in rural areas of the U.S. must drive a long distance to receive their medical treatments. According to Yahoo News, twenty-nine-year-old Leah Stavenhagen, now living and…
The recent FDA approval of Vyvgart (efgartigimod) for generalized myasthenia gravis (gMG) marks the first drug approval for Argenx. Vyvgart is designed specifically for adults who test positive for…
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