Updates from the Phase 3 clinical trial called FIGHT-302 were just released at the 2021 European Society for Medical Oncology World Congress on Gastrointestinal Cancer. The trial is comparing two…
A survey of oncologists has found that more education is clearly needed regarding cholangiocarcinoma. Specifically, more education would be beneficial regarding testing and targeting the mutations which cause the condition…
Bacterial meningitis is a severe condition that is linked to high mortality. Unfortunately, newborns are much more susceptible to bacterial meningitis. In fact, this infection occurs more than 30 times…
Becca Meyers, a 26-year-old swimmer who was born deaf, has just been told she can't bring her care assistant to the Tokyo Games. As a result, she has had to…
Researchers from Boston University and Boston Medical Center have recently uncovered that base editing is able to correct the gene mutation which causes alpha-1 antitrypsin deficiency (A1AD). This condition impacts…
A study conducted by researchers at Nagoya University in Japan and researchers from Yale University in the United States was recently published in the European Respiratory Journal. This study utilized single-cell…
Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within…
Dr. Jerry Vockley from the UPMC Children's Hospital of Pittsburgh has been studying the rare disease VLCAD for years. His work has led to the creation of two therapies which…
Salarius Pharmaceuticals has just announced the addition of a new clinical trial site for their study investigating seclidemstat as a treatment for Ewing sarcoma (relapsed or refractory) as well as…
As we've learned more about individuals facing long-term symptoms from COVID-19, we have heard more and more about how it mimics other chronic conditions. For instance, those suffering from chronic…
Many individuals in our day and age find it difficult to sleep. Although the first humans awoke with the sun and slept with the moon, we don't operate that way…
Chronic Fatigue Syndrome Chronic fatigue syndrome (CFS), otherwise known as myalgic encephalomyelitis (ME), is a rare disease primarily characterized by unexplainable and intense fatigue that is not relieved with rest.…
A new clinical trial is starting for Friedreich's ataxia (FA). This trial will investigate if supplementation of vitamin D in its active form, also known as calcitriol, will aid in…
A new study, funded by the NIH and conducted by Vanderbilt University Medical Center (VUMC), is working to assess allergic reactions to COVID-19 vaccines. The trial will include individuals between…
Two new reports of Guillain-Barré syndrome (GBS) have been documented in response to the AstraZeneca COVID-19 vaccine. Previously, published papers have documented 4 cases in England and 7 cases in…
Auto-Brewery Syndrome Auto-brewery syndrome is also known as gut fermentation syndrome. It occurs when fungi or bacteria within the gut produce ethanol by endogenous fermentation. Several different strains may be…
Aurinia Pharmaceuticals has just announced that Otsuka Pharmaceutical Europe Ltd. (OPEL), their licensing partner, has just filed a Marketing Authorization Application for an investigative therapy for lupus nephritis (LN) called…
I-Mab has just announced China's Center for Drug Evaluation has officially approved the new drug application for felzartamab as therapy for systemic lupus erythematosus (SLE). That means that a Phase…
BioMan Pharmaceuticals has just announced that vosoritide has just received a positive opinion from the Committee for Medicinal Products in Europe, which means marketing authorization is recommended. Further, it has just…
The very first corneal allogenic intrastromal ring segment surgery (CAIRS) for keratoconus has just been completed in Australia. It was completed at Queensland Eye Institute by David Gunn. This procedural…
A child's life was recently saved thanks to ultra-ultra-rapid genome sequencing which provided him his rare disease diagnosis in just 13 hours. The Human Genome Project began 30 years ago…
Jo Blaney was diagnosed with cyclic vomiting syndrome when she was pregnant with baby number four. She is now striving to bring awareness to the condition, which impacts patients' daily…
Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…
Impel NeuroPharma has been working on a campaign focused on migraines. It is called Reroute Migraine Relief, and it was built in coordination with an artist who suffers from migraines…
Strongbridge Biopharma has just announced that the FDA has decided to review the New Drug Application for RECORLEV, a therapy for endogenous Cushing's syndrome. The target action date is January…
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