Pfizer’s Regional President for Rare Disease, Reda Guiha, recently offered his opinion to The Parliament magazine about the urgency of what he refers to as the ‘rare disease crisis.’ Pres.…
A recent study investigating treatment for adult acromegaly patients has revealed that switching from injectable somatostatin receptor ligands to oral octreotide leads to an improvement in quality of life and…
Have you ever heard of base editing? This relatively new technology within the field of genome editing offers the potential opportunity to treat a variety of genetic conditions. According to…
ACADIA Pharmaceuticals has released data on NUPLAZID, a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors. This treatment is meant to treat Parkinson's disease patients, specifically the delusions…
According to a story from bbc.com, five month old Arthur was born with the rare disease spinal muscular atrophy (SMA). He is one of the first people in the UK…
According to a story from MedPage Today, a recent study has determined the familial hypercholesterolemia (FH) can often go undetected on genetic tests that rely on arrays. As an example,…
Intravenous immunoglobulin, or IVIG, is a therapy used for an array of autoimmune conditions. It has anti-inflammatory properties which help support many diseases. Recently, researchers have demonstrated the potential of…
Written by Kevin Schnurr A renal patient seeks clarity amidst ongoing, conflicting reports. It’s not every day I’m constantly reminded of my status as someone post-organ transplant, but…
70-year-old Trevor Hardie from New South Wales is recovering after almost losing his life to leptospirosis, a bacterial infection that is spread through the urine of mice. He's not 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…
In 2020, COVID-19, a novel coronavirus, began impacting the globe. Caused by the SARS-CoV-2 virus, COVID-19 has resulted in 172 million diagnoses and 3.7 million deaths worldwide. While acute kidney…
Medical professionals are still trying to understand all of the underlying causes of Parkinson's disease, a progressive disease that sees the degeneration and death of motor neurons. A recent study…
Early on in 2021, the FDA approved voclosporin as an add-on treatment for patients with lupus nephritis (LN), a frequent complication of lupus. Ultimately, this approval was a positive decision,…
Altogether, AB Science has been evaluating a tyrosine kinase inhibitor, masitinib, for a variety of conditions including amyotrophic lateral sclerosis (ALS), COVID-19, and mastocytosis. But according to ALS News Today,…
Rare disease research is often lacking due to a lack of funding, interest, or awareness. To combat this, the Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have joined…
According to a story from the Green Bay Press Gazette, the beginning of summer is rapidly approaching and with warming temperatures comes the emergence of ticks. These little parasites are…
A press release by MYND Sciences announced that the drug research and development company, MYND Life Sciences Inc. has their new intellectual patent for their method of finding biomarkers to…
According to an article in the Times Union, a group of parents with children who have been diagnosed with rare diseases are leading the crusade to foster federal legislation which…
Sobi as well as the Hellenic Institute for the Study of Sepsis have just announced successful results from their SAVE-MORE study for those with COVID-19. The results were from 28…
In a recent news release on Morningstar, biopharmaceutical company Albireo Pharma, Inc. ("Albireo") shared that its investigational drug candidate Bylvay (odevixibat) earned a positive opinion from the European Medicines…
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…
Sometimes, medical studies just don't go the way that companies want them to. According to MedCity News, this is what biotechnology company Larimar Therapeutics ("Larimar") was faced with during…
In 2019, generic pharmaceuticals and biosimilars leader Sandoz launched intravenous treprostinil for patients with pulmonary arterial hypertension (PAH). But according to a new media release, generic treprostinil is now…
Cure Rare Disease is a nonprofit organization that aims to help those who are impacted by rare diseases. Currently, the Boston-based company is planning a fundraiser to celebrate all of…
When Gwen Dillon's son, Aaron, was just 1.5 years old, he was diagnosed with craniosynostosis, a birth defect in which the bones of a baby's skull close early. According to…
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