According to a story from businesswire.com, the pharmaceutical company Bayer has recently announced the release of results from its phase IV trial. This trial evaluated the effects of the drug…
Leigh syndrome is a rare mitochondrial disease that is not yet well understood by medical professionals. When this lack of comprehension exists, it is impossible to create treatments or improve…
According to a story from PR Newswire, the biotechnology company Ionis Pharmaceuticals recently announced promising findings from its phase 2 clinical trial. This clinical trial was evaluating the company's experimental…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
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…
Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich's ataxia (FA). This study showed that the therapy resulted in…
Over the next 2-2.5 years, biotech spin-out company Augustine Therapeutics will be working to understand both the underlying biology of Charcot-Marie-Tooth disease (CMT) and potential therapeutic options for patients.…
According to a study from hindawi.com, the serum glycocholic acid (GCA) to total bile acid (TBA) ratio can be used to identify people living with nonalcoholic fatty liver disease (NAFLD).…
Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…
Charcot-Marie Tooth disease (CMT) may be the most common inherited disorder of the peripheral nervous system, but that only puts the official patient count in Denmark at about 22.5 cases…
At the Endocrine Society's Annual Meeting, or ENDO 2021, Amolyt Pharma ("Amolyt") shared positive clinical data from a Phase 1 clinical trial evaluating AZP-3601 for patients with hypoparathyroidism. Although…
According to a story from Medical Xpress, an uncommon fungal infection has been discovered in Alberta, Canada. This infection, called histoplasmosis, is transmitted through the fecal matter of bats and…
A recent study published in Arthritis Care & Research illustrated how higher dosages of prednisone lead to better outcomes for lupus nephritis (LN) patients. After one year, participants were found to…
In a study published on pubmed.gov, an investigation of 100 patients living with progressive supranuclear palsy, a rare disorder, has helped to confirm the usefulness of a diagnostic approach called…
A Phase 3 clinical trial run by Pharnext for Charcot-Marie-Tooth disease Type 1A (CMT1A) has just been awarded 13.3 million dollars. $7.2 million comes from existing shareholders and $6 million comes…
Rare disease therapies have an exorbitant cost. Fewer patients means higher costs in order to support the development of treatments. In the United States, 966 billion was spent in 2019…
Chronic kidney disease (CKD) affects hundreds of millions of people throughout the world. As of 2017, 697.5 million people were estimated to have CKD by this study published in Nature Reviews…
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Rare diseases are much more common than people think. The word "rare" is off-putting. But the word only pertains to each individual rare condition. Collectively, rare diseases are quite common.…
As the pandemic shifted cocktail hour onto zoom and professors posted their full course material online, many rare patients reveled. Not because we weren’t suffering through the pandemic too, but…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
In a recent press release, biopharmaceutical company Aeterna Zentaris Inc. shared that the company would be developing a new therapy for patients with hypoparathyroidism. The preclinical development program will focus…
Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in…
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…
A new study is in the works, and it will investigate how cystinosis, a genetic disorder characterized by an accumulation of the amino acid cystine in the cells, leads to…
The majority of Parkinson’s patients treated with Levodopa experience relief from the major motor effects of Parkinson’s. However, according to a recent article in Biospace, after prolonged use of Levodopa…
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