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…
According to a recent article, $100 million has been given to four research teams by the Cancer Grand Challenges program for cancer research. Cancer Grand Challenges Program The Cancer…
The novel program called BeginNGS™ was described in a recent article which has a goal of testing for five hundred disorders and several thousand infants. Rady Children's Institute for…
According to a story from Globe Newswire, the global healthcare company Novo Nordisk recently announced encouraging findings from its phase 3 clinical trial. This study was evaluating somapacitan (marketed as…
Genetic testing has always been costly and directed at DNA sequencing. Only a limited number of genes could be tested. It was useful to a small number of patients…
Unfortunately, it can sometimes be difficult to get companies to perform research and develop therapeutics for rare diseases, but the FDA came up with a process to spur research…
Every year, June 30 is marked as Arthrogryposis Awareness Day. It was created by Ani Samargian, the founder of Arthrogryposis Multiplex Congenita Support Inc., back in 2005. The day is…
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…
According to a recent news release from PR Newswire, the European Medicines Agency (EMA) has recently awarded elamipretide its Orphan Drug designation for the treatment of Barth syndrome. This treatment,…
When Ivy Reed was just two weeks old, she was first diagnosed with two conditions: non-verbal autism and propionic acidemia, the latter of which is a rare metabolic disorder. Because…
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…
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…
According to an article recently published in Targeted Oncology, the FDA approved olaparib (Lynparza), a product of AstraZeneca Pharmaceuticals, in March of this year. Lynparaza will be used as adjuvant…
June 30, 2022 will be recognized as Arthrogryposis Multiplex Congenita (AMC) Awareness Day, a time to spread awareness about arthrogryposis multiplex congenita among the general public and in the medical…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare or life-threatening diseases or conditions. Rare conditions are those affecting…
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…
Don't forget to take a look at Part 1 of our interview, where Jessica discussed her trigeminal neuralgia journey, what this condition is, and why she decided to start the…
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…
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,…
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