Blueprint Medicines Corporation has just announced that they have submitted a supplemental New Drug Application (NDA) to the FDA. This application is for a therapy called AYVAKIT, which was developed…
According to European Pharmaceutical Review, results from a Phase 1/2a clinical trial support the use of AstroRx for patients with amyotrophic lateral sclerosis (ALS). The clinical trial, headed by Kadimastem…
A study published in BMC Pulmonary Medicine focuses on the role of endothelial progenitor cells and fibrocytes in lung sarcoidosis. They specifically investigated how these cells, along with CD34 + cells, spurred…
Researchers have uncovered a new way to treat cancer that could lessen the side effects that patients often experience from chemotherapy. At the same time, it improves the curative power…
No deal Brexit means that the UK will be excluded from the 24 ERNs (European Reference Networks). Without the sharing of information, data, and research across countries, rare disease research…
According to OncLive, Oncolytics Biotech and Roche are pairing up to run a new German Phase ½ clinical trial. The GOBLET trial will evaluate pelareorep in conjunction with atezolizumab (Tecentriq)…
In a recent press release, biopharmaceutical company Rhythm Pharmaceuticals (“Rhythm”) announced positive data from a Phase 3 clinical trial. During the trial, Rhythm evaluated setmelanotide for patients with Bardet-Biedl syndrome…
In a late December 2020 press release, biopharmaceutical company Cerecor Inc. (“Cerecor”) announced the FDA acceptance of an Investigational New Drug Application (IND) for CERC-007. The human monoclonal antibody (mAb)…
Have you ever had a feeling that you knew something was off, but you just didn't know what? That is how I felt for more than half of my life.…
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…
Pulmonary fibrosis is a condition that is caused by scarring of the lungs which can lead to shortness of breath and other respiratory issues. Some cases of COVID-19 have lead to…
According to GlobeNewswire, Health Canada has approved of a biosimilar, Hyrimoz, to treat ankylosing spondylitis and eight other rare conditions. Sandoz Canada is excited about this approval, as it expands…
Many rare disease patients understand the financial burdens of living with a rare disease. As the therapies are only supporting a small number of patients, the price tag is often…
Complex Regional Pain Syndrome Complex regional pain syndrome (CRPS) is a rare condition that causes prolonged pain after a minor injury. This pain is severe and can be debilitating. One…
The FDA has recently approved of GlaxoSmithKline's (GSK) treatment for lupus nephritis (LN), BENLYSTA. This therapy is the first of its kind, meant to treat systemic lupus and adults with…
Could the perennial thunder god vine, usually found in China and Taiwan, be aptly named? Does it provide a strong and beneficial impact? According to Medical Xpress, this vine, also…
Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo's mission,…
As the pandemic has raged on, we have learned more and more about what side effects can occur from severe COVID-19 infection. Some of these are common in patients with…
The Rare Disease Legislative Advocates (RDLA) held a webinar on December 3rd, 2020. This event focused on the organization's goal of passing Cures 2.0, a bill that would help further…
In a recent press release, pharmaceutical company Acer Therapeutics, Inc. ("Acer") announced the publication of data on celiprolol for patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS). The article, published in the European…
Researchers have uncovered that there is a link between Alzheimer's disease and three eye conditions- glaucoma, diabetic retinopathy, and macular degeneration (age-related). This could mean that there may be another…
There is a large stigma against those with disabilities. Many misunderstand the wide diversity in disability and the success that those who are disabled can have in their lives. This…
Before 1983, there were few treatments for rare diseases. Various obstacles stood in the way, including little representation for patients, few incentives, and a lack of resources. Fortunately, the Orphan…
Yash Cary knows what it's like to spend the holidays in the hospital. In fact, three of his last six Christmases have been at UNC Hospital. This is because Yash…
A team of researchers from the Walter and Eliza Hall Institute of Medical Research (WEHI) in Australia recently made strides in stomach cancer research. While searching for a better understanding…
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