According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…
Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…
The receptors in the human epidermal growth factor receptor 2 (the HER2 ) gene are active participants in determining whether healthy breasts can repair themselves. According to a recent article…
Living with an invisible illness can be incredibly difficult. Those around you do not understand the struggle, and they can even discount your illness. Women's Health talked to three women…
CIDRAP recently featured JAMA NetworkOpen's online reports of two timely research letters. The letters outlined the complications that may arise for aortic stenosis patients who have had to delay transaortic valve…
Researchers have recently conducted a study using mouse models of Charcot-Marie-Tooth (CMT) disease, titled "CMTM6 Expressed on the Adaxonal Scwann Cells Surface Restricts Axonal Diameters in Peripheral Nerves." It was…
Waking up to the news has one encouraging note, another new day and another new drug is being tested. According to an article in Biospace, the news from Sorrento Therapeutics…
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…
As published in a press release by the Hereditary Neuropathy Foundation, a project funded by the National Institutes of Health (NIH), researchers at the University of Rochester, the University of Pennsylvania, and…
The FDA has recently granted the Fast Track designation to AGLE-102, a treatment for dystrophic epidermolysis bullosa. This designation will allow for the treatment to reach patients at a quicker…
When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting…
Near the end of September, Hansa Biopharma ("Hansa") announced promising data from a Phase 2 clinical trial on imlifidase. This therapy is designed to treat patients with severe anti-GBM antibody…
As reported recently in SciTech Daily, for years scientists have recognized red blood cells (RBC) as a vehicle to deliver drugs within the human body. They circulate for days and…
As reported in Cystic Fibrosis News; the 2020 Santander Universities Entrepreneurship Awards are part of an initiative to encourage young entrepreneurship via support of student led startups. This year there…
As reported in Newswise, a new experimental study on vaping, a popular electronic device used to consume nicotine or cannabis, has found that the e-cigarette products with nickel-chromium alloy heating…
According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…
According to a recent report in the Journal of Clinical Oncology, over one thousand patients with high-risk stage III melanoma were included in the Keynote-054 clinical trial (NCT02362594). The…
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…
At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
Happy Friday! October is Dysautonomia Awareness Month. Dysautonomia is a collection of conditions that affect the autonomic nervous system. This can lead to a loss of control of the body's…
Owlstone Medical recently announced the success of its patient study demonstrating its Breath Biopsy®, using limonene as a biomarker in measuring liver function. The study demonstrated how exhaled limonene, a…
A research team from IPM.ai, a subsidiary of Swoop, and Insmed, a biotechnology company, recently collaborated on a case study that demonstrated how AI and machine learning could be used…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
Historically, clinical trials have played an immense role in collecting patient data, understanding disease progression, and crafting more targeted treatments. This is especially true in the oncology realm. But according…
PRNewswire recently carried BERG’s announcement of the results of its COVID-19 clinical study. BERG, a biotech company based in Framingham, Mass., utilizes artificial intelligence in its research of diseases and…
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