In order to create treatments and diagnostic methods for rare diseases, it's necessary to have a thorough understanding of said disease. A recent study aimed to do just this. Titled…
On March 29, 2022, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar titled "An Overview of Amyloidosis and WM with Dr. Morie Gertz." The goal of this program was…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
An article recently highlighted the stories of young patients with Ehlers-Danlos syndrome and how they are taking their diagnoses to spearhead research about the rare disease. Ehlers-Danlos Syndrome (EDS) Ehlers-Danlos…
In the past, filgotinib (Jyseleca) has largely been used to treat patients with rheumatoid arthritis (RA). However, researchers hypothesized that the treatment could be beneficial in other indications. Following…
The COVID era emphasized the importance of maintaining a strong immune system. But what about people who are less fortunate? Johns Hopkins researchers recently announced some good news for…
We often think of cyclic vomiting syndrome (CVS) as a rare disease, but is this really the truth? According to a recent article from New Castle News, this is not…
In the past, there has never been an approved therapeutic option for those with acid sphingomyelinase deficiency (ASMD). This meant that there was a huge unmet need within this patient…
Roger Bliss, 68-year-old pulmonary hypertension (PH) patient, was diagnosed with chronic lymphocytic leukemia and small lymphocytic lymphoma. In addition, Roger has celiac disease. He recently interviewed with Pulmonary Hypertension News.…
For the first time, a team of researchers measured heart rate variability (HRV) in those with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) to understand how the autonomic nervous system (ANS) responded…
On March 24, 2022, the Rare Disease Legislative Advocates hosted its monthly webinar to provide updates on legislation and public policy that is relevant to the rare disease patient community.…
A recent article discusses the various relationships between osteoporosis, osteonecrosis, and bisphosphonate medications. Osteoporosis (Paget’s Disease) Osteoporosis causes bones to become weak and brittle — so brittle that a fall…
Pancreatic cancer is typically a difficult cancer to treat, leading to poor outcomes and high mortality rates. One of the reasons for this is the difficulty of diagnosis; it often…
According to a recent article, researchers have found that a sensitivity to sounds such as chewing or breathing may actually be caused by misophonia. Misophonia Misophonia, which literally translates to…
Sparsentan is a dual endothelin angiotensin receptor antagonist (DEARA) currently being developed for the treatment of IgA nephropathy (IgAN). The newest update on sparsentan was recently shared by Travere Therapeutics via…
Mitochondria are called the ‘batteries’ that send power to our body’s cells. They convert energy from food into forms that cells can utilize. Mitochondria contain 0.1% of mitochondrial DNA (mtDNA)…
For many rare diseases, the symptoms and experiences of patients are variable. It's extremely rare - if possible at all - to see a rare disease that can be be…
According to a recent article, the Hamoui Foundation and LUNGevity Foundation granted $1 million to three researchers in order to study RET-positive lung cancer. RET-Positive Lung Cancer There are two…
According to an article recently published in Business Wire, Global Genes, a leading advocacy group, released a progress report on rare diseases. The report includes data and studies as…
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…
Note: this story was originally published by HAE Junior, a Patient Worthy partner Interview with Malena Vetterli from the European Reference Network (ERN-RITA) for rare immunodeficiencies, autoinflammatory and autoimmune diseases.…
When Melinda Jansen van Vuuren was 13 years old, she began smoking cigarettes. For years after, Melinda smoked up to 15 cigarettes daily. Although she has since quit smoking, Melinda…
Munique Slongo is a rare disease patient diagnosed with Fabry disease. She has taken the time to share her story about what life with this diagnosis has been like. Fabry…
Previous studies have shown that preterm infants (babies born before 37 weeks of pregnancy) often have vitamin D insufficiency. According to Medical Dialogues, researchers in Korea sought to understand the…
Without treatment, an estimated 20% of those with progressive keratoconus may eventually require corneal transplantation. Glaukos, a company creating novel therapies for chronic eye diseases, developed the iLink corneal cross-linking procedure to…
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