At the moment, there are two FDA-approved treatment options for people with idiopathic pulmonary fibrosis (IPF). However, given the needs of patients, it is necessary to develop additional therapeutic…
Clinical trials are a crucial part of learning more about certain diseases, as well as better understanding the impact of various therapeutic options. For example, a clinical trial can…
The current standards-of-care for those with Duchenne muscular dystrophy (DMD) include corticosteroids, immunosuppressive treatments, physical therapy, and occupational therapy (among others). Gene therapy has the potential to change the treatment…
Diffuse midline glioma (DMG) refers to a rare and extremely aggressive brain tumor that is most common in children. Chemotherapy and radiation are the current standards-of-care. Unfortunately, even with…
Are there therapeutic options for patients living with unresectable (inoperable) pancreatic cancer? According to an article in MedScape, researchers sought to understand whether sunitinib or a combination of peptide receptor…
In the past, there have been a small number of studies exploring the connection between sickle cell disease (SCD) and craniosynostosis. More recently, researchers sought to understand whether there was…
Research has shown that up to 70% of individuals who undergo stem cell transplantation experience organ damage within just a few months. Many develop graft-versus-host disease (GvHD), a complication in…
Cushing’s disease (or Cushing disease) occurs when a benign pituitary growth or tumor leads to the overproduction of adrenocorticotropic hormone (ACTH). As ACTH is overproduced, the body also begins…
In the medical world, it’s no secret that misdiagnoses happen. But these misdiagnoses can be costly; patients may miss out on earlier treatment and prevention, and misdiagnosis may correlate with…
Currently, there is no known cause for multiple sclerosis (MS). While many doctors consider MS to be an autoimmune disease, nobody knows what prompts this immune response. A combination…
According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…
Both Libtayo (cemiplimab) and Keytruda (pembrolizumab) have been shown to be beneficial treatment options for patients with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). But could Opdivo (nivolumab)…
Currently, there are a number of therapies which can be used to treat individuals with dermatomyositis: corticosteroids, immunosuppressive agents, intravenous immune globulin (IVIG). Typically, corticosteroids are considered a first-line treatment,…
Scientists affiliated with Massachusetts General Hospital (MGH) and Children's Hospital Philadelphia recently identified a novel mitochondrial disorder in a pair of identical twins. Doctors began studying these twins when…
Proton pump inhibitors (PPIs) are a class of medicine that reduce the amount of stomach acid production. They are commonly known for their use in acid-related disorders, such as gastroesophageal…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. A rare disease is one…
Have researchers from Rice University and Baylor College of Medicine discovered a potential treatment option for mesothelioma? According to a news release from the U.S. National Science Foundation, who…
Researchers have been evaluating '1104, an investigational immune-resetting molecule, for patients with eosinophilic esophagitis (EoE). Through immune resetting, some believe that '1104 could reduce the inflammatory cascade related to…
According to an October 31, 2022 press release from muscle disease company Dyne Therapeutics, Inc. ("Dyne"), the company's therapeutic candidate DYNE-251 earned Fast Track designation from the FDA for Duchenne…
Over the past decade, there has been growing research into the effects of various chemicals, like herbicides or pesticides, on human health. Researchers want to understand how herbicide or pesticide…
An estimated 4 million adults within the U.S. have fibromyalgia, a chronic disorder characterized by fatigue, problems with sleep and memory, and musculoskeletal problems. Prior studies have found that a…
Over the years, there has been more research into the impact of diet and nutrition on our overall health. How does what we eat – and how it was prepared…
Is there any relationship between achalasia and esophageal cancer? According to Healio, a research study found that there does seem to be some level of correlation. In Abstract S388, presented…
From October 7-11, 2022, the AAPNational Conference & Exhibition offered insight into clinical practices, trends, and other important information within the pediatrics specialty. According to Contemporary Pediatrics, the Family Heart…
The current standard-of-care for Fabry disease is called enzyme replacement therapy (ERT). According to Fabry Disease News, researchers have been working to evaluate an experimental ERT for patients called ISU303.…
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