In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which…
Recently diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations now have a new treatment option, as the FDA has granted approval to TIBSOVO (ivosidenib tablets)! This is an expansion…
Personable. Loving. Impactful. Those are all words that Angie Kane would use to describe her son Colton, who passed away at 21 years old in October 2021. When Angie talks…
We have been told occasionally that certain phrases we use when speaking with a terminally ill patient may sound insensitive. We have also been told what we should not say…
Long-term extension (LTE) data is now available for setmelanotide, which is a treatment for rare causes of obesity, such as Bardet-Biedl syndrome (BBS) and POMC or LEPR deficiency. Data on…
For the last two and a half years, the COVID-19 pandemic has dominated the headlines. It has been hard to look online – or do anything in person – without…
Pancreatic cancer is one of the deadliest forms of cancer. According to the American Cancer Society (ACS), only about 11% of patients are expected to survive more than five years…
Lately, the first thing that enters a person’s mind if they encounter an unusual pain is that they may have cancer. Yet that was the last thing Georgina Masson, an…
Advocacy is an extremely important part of supporting and helping the rare disease community. A big part of this is rare disease advisory councils (RDACs), which are governmental groups comprised…
According to a recent article, popular Filipina television host, also known as the Philippines' “Queen of all Media," has been diagnosed with Churg-Strauss Syndrome. Churg-Strauss Syndrome Churg-Strauss syndrome, or eosinophilic…
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…
As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
Jeremy Schreifels was just a young child when he was first diagnosed with Tourette syndrome, a nervous system disorder characterized by sudden, involuntary movements or vocalizations (“tics”). In Jeremy’s case,…
Sydney Miller, born in the UK in March 2020, has an extremely rare disease. Newsweek recently carried a story about Sydney and her illness. This little girl captured the hearts…
It is very rare that one might say that a car accident saved their life – but Yolanda Carusciello, who lives in Brooklyn, can say just that. Of course, when…
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…
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…
Leukemia, in general terms, is a form of cancer that affects the bone marrow and blood cells. According an article in Medical News Today, leukemia is categorized in accordance…
During this month, the Dravet Syndrome Foundation (DSF) organizes a variety of activities and resources to help raise awareness for Dravet syndrome, a rare form of epilepsy. They make it…
If you've been paying attention to the news lately, you know there's a shortage of baby formula in the United States. Many, many mothers and children have been affected, leaving…
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…
If you've been paying attention to the news lately, you've probably heard of a rare viral illness that has been spreading around the globe: monkeypox. While monkeypox is more common…
Having lived over 20 years with chronic pain from dystonia (a neurological movement disorder) in order to best manage my condition, I have to be very mindful of the way I live…
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