For months, those within the rare disease community have been watching to see whether Upstaza (eladocagene exuparvovec) would be approved for use within the European Union. In fact, in…
A Phase 2 clinical trial which sought to evaluate the safety, efficacy, and tolerability of NP-120 (ifenprodil) for idiopathic pulmonary fibrosis (IPF) had some surprising results. Initially, the study…
Clinical trials are an important tool in medical research to understand more about the safety, efficacy, and tolerability of therapeutics - and how these may influence patient outcomes. Recently,…
In a recent news release, clinical-stage biotechnology company Amolyt Pharma ("Amolyt") shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought…
A few years ago, Dr. Thomas Weimbs, a researcher at the Weimbs Lab of UC Santa Barbara, performed a study on mice models of polycystic kidney disease (PKD). During…
Recently, the International Society on Thrombosis and Haemostasis (ISTH) held its 2022 Congress from July 9th to 13th. During the conference, researchers and other stakeholders discussed research, care, and…
Earlier this year, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its International Congress. During the Congress, researchers discussed the latest in research and scientific innovation within…
Currently, there are few therapeutic options for people who become infected with the Nipah virus. Rather, treatment is symptomatic. However, that may soon be changing. According to the European…
Early diagnosis and treatment is crucial for patients with primary hyperoxaluria type 1 (PH1). Unfortunately, due to a high prevalence of misdiagnosis, as well as poor therapeutic access in…
Medical research and clinical trials are extremely important tools in learning more about specific diseases, as well as understanding and evaluating potential treatments. For example, the Phase 2 VIBRANT…
Rituximab has been used to treat patients with cancers such as non-Hodgkin’s lymphoma (NHL), and various autoimmune diseases. Recently, a study sought to understand whether rituximab treatment would be safe…
Sometimes, certain health conditions can increase the risk of developing other conditions. According to a new research study, this may be the case with porphyria and hepatocellular carcinoma. An article…
On July 7, 2022, biotechnology company Akari Therapeutics, Plc ("Akari") shared via news release that the first patient in its clinical trial has completed their course of treatment. Within…
In a news release, biopharmaceutical company Fulcrum Therapeutics, Inc. ("Fulcrum") shared that the first patient was dosed in the Phase 3 REACH study. Within the study, researchers are evaluating…
In a press release on the company website, biopharmaceutical company AbbVie shared that preliminary data was available regarding navitoclax, an investigational therapy, for individuals with primary myelofibrosis. The data…
From May 13-18, 2022, the American Thoracic Society (ATS) held its Annual Meeting, where researchers and stakeholders discussed research, patient care, and public health trends within the fields of…
In many cases, orally administered PDE5 inhibitors – such as Cialis, Levitra, and Stendra – have been used to treat erectile dysfunction. These vasodilating drugs were found to be…
The number of Alzheimer’s disease diagnoses has been rising rapidly over the last few decades. Today, an estimated 6.5 million Americans alone are living with this disease. Yet there…
The American Society of Clinical Oncology (ASCO) Annual Meeting (“ASCO 22”) took place this year from June 2nd through the 6th, 2022. During this Annual Meeting, stakeholders within the…
Sometimes, doctors choose to treat a certain condition with a specific medicine or class of medicines. But what happens when that medicine causes unintended and potentially harmful reactions? For…
The last approved treatment option for Pompe disease in Europe was given approval in 2006, so 16 years ago. However, the European Commission (EC) recently approved a new therapeutic…
Sometimes, it can be difficult to diagnose amyotrophic lateral sclerosis (ALS), a progressive neurological disease which causes nerve cell (motor neuron) death in the brain, brain stem, and spinal…
IBD (inflammatory bowel disease) is an umbrella term for ulcerative colitis and Crohn’s disease involving chronic inflammation in the digestive tract. Common symptoms are fatigue, pain, and reduction in appetite.…
When it comes to cancer treatment, it's important to understand what leads to improvements in survival; knowing these things helps to provide the best outcome for the patient. That's why…
Over the last few decades, there have been immense advancements in medical technology and care options. However, not all of these advancements contribute to better patient outcomes. For example, more…
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