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,…
Before you read Part 2 of Lindsey's interview, make sure to go back and take a look at Part 1! In Part 1, we discussed Finn, the diagnostic journey for…
The medical term for an “informal caregiver” describes a person who willingly takes the responsibility to help a spouse, family member, friend, or partner. The level of care and responsibility…
Within the United Kingdom, there's a program titled the Innovative Licensing and Access Pathway (ILAP), which is meant to give patients earlier access to developing treatments. The first step to…
Altogether, an estimated 15-30% of people around the globe have nonalcoholic fatty liver disease (NAFLD), a condition in those who drink very little or not at all which mimics liver…
When you think about a visit to the dentist, it may – at first – seem innocuous, something that you need to take care of for your health. But for…
The EULER 2022 Congress took place this year from June 1st-4th, 2022. During that time, rheumatology professionals met to discuss trends, guidelines, and patient care within the field of rheumatology.…
For as long as she can remember, Destiny Fiaschetti has been incredibly committed to becoming a foster parent. She dreamed of providing a home for children in need, especially those…
On June 10, 2022, Patient Worthy partner GACI Global hosted an hour long webinar on the subject of hearing loss, featuring Dr. Karen Wilber. GACI Global is a nonprofit organization…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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