According to a recent article, PharmaDrug Inc. revealed that their study of combining cepharanthine and chemotherapy to treat IND-enabling esophageal cancer has yielded positive results. Esophageal Cancer Esophageal cancer occurs…
This year, the National Lipid Association held its Scientific Sessions from June 2-5, 2022. During the Scientific Sessions, attendees discussed new research, clinical best practices, and other topics within this…
A recent study - which you can find in the journal Muscle & Nerve - investigated Soliris as a treatment for anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG). It…
Liver cirrhosis (scarring) can be fatal because damage to the liver is irreversible. A cirrhotic liver leads to liver failure, liver cancer, and eventually death. According to a report in…
Did you know that atrial fibrillation (AF) increases the risk of a stroke by 3 to 5 times? However, blood thinners such as warfarin, or other potential treatments, can protect…
Clinical trials are useful not only for initially establishing the efficacy of treatments for rare diseases, but for demonstrating that these results are durable as well. The MPOWER trial, which…
Understanding how a patient's condition will progress is extremely important in providing them with the best treatment, and autosomal dominant polycystic kidney disease (ADPKD) does not stray from this trend.…
There is no doubt that artificial intelligence (AI) is going to change the future of healthcare. AI can be used in disease prediction, treatment, identification, diagnosis, and management. For example,…
According to a recent article in The British publication The Economic Times, a group of researchers at McMaster University have a win-win proposal for myotonic dystrophy (MD) patients. Eleven people were…
The future of healthcare is going to be changed and impacted by the use of artificial intelligence (AI), hopefully in beneficial ways. According to an article from McMaster University, researchers…
This year, the Congress of Clinical Rheumatology East took place from May 12-15, 2022. During the conference, clinical rheumatologists gathered to discuss trends in rheumatology, practice guidelines, and cutting-edge therapeutics.…
Inherited retinal diseases (IRDs) are a group of genetic ocular disorders characterized by vision loss or blindness. A few examples are retinitis pigments (RP) and Usher syndrome. There has been…
From June 11th until the 14th, the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting was held in Vancouver, Canada. At this meeting, Attralus Inc. shared new data…
In clinical trials evaluating Zolgensma for children with spinal muscular atrophy (SMA), the maximum weight of participants was 18.7 pounds. However, parents with children weighing over that weight might be…
In normal development, genes are usually inherited in pairs - with one copy from the father and one inherited from the mother. When one copy of the TCF4 gene, which…
Anemia is a common symptom for chronic kidney disease (CKD), but there hasn't been much research done on the association between the two or the adverse outcomes which ensue as…
In the past, research has shown that bleach baths can be a helpful tool in addressing or relieving symptoms associated with dermatological issues. For example, some doctors suggest the use…
Have you ever heard of azithromycin? It’s an antibiotic. Chances are, if you’ve ever had a bacterial infection, you’ve even taken it. But according to Medical XPress, researchers are currently…
Earlier this year, the U.S. Food and Drug Administration (FDA) approved mitapivat (PYRUKYND) for the treatment of patients with hemolytic anemia due to pyruvate kinase deficiency (PKD). According to 2…
This year, the American Academy of Dermatology (AAD) Annual Meeting took place from March 25-29, 2022. During the meeting, researchers and stakeholders discussed trends and research within the field of…
What type of cognitive changes are present in individuals with spinal muscular atrophy (SMA) type 3? More so, do these cognitive changes affect clinical factors and, if so, how? According…
The Irish Times recently featured a book by Dr. Karl Herrup about “How Not to Study a Disease." Dr. Herrup is clear that he opposes the theory that the amyloid…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare conditions. A rare disease or condition is defined as…
In the past, there have been a variety of research studies exploring and working to learn more about cystinuria, a rare genetic condition causing cystine, an amino acid, to accumulate…
Sometimes, it can be difficult to prompt the research of and creation of therapeutics for rare diseases. This is one of the reasons why the FDA created its Orphan Drug…
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