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…
It is no secret that certain conditions can sometimes be debilitating or make it harder to partake in day-to-day life. According to Hypoparathyroidism News, researchers recently sought to understand the…
An article recently came out highlighting one young woman’s struggle with the rare cancer rhabdomyosarcoma. Rhabdomyosarcoma Rhabdomyosarcoma is a rare form of cancer that develops in soft tissue. It…
Recent clinical trials have proven that treatment for relapsed ovarian cancer is not a standard “one size fits all." After a relapse, areas where tumors spread and grow in the…
Past research has shown that rogue Th17 cells could help spur the manifestation of autoimmune conditions like multiple sclerosis (MS). But why do these cells go wrong and how could…
From January 20th through the 22nd, 2022, researchers, specialists, and other stakeholders descended on the Gastrointestinal Cancers Symposium to discuss new and innovative research on gastrointestinal cancers. During the Symposium,…
A recent study has demonstrated that neuromyelitis optica spectrum disorder (NMSOD) patients treated with eculizumab have a lower risk of adjudicated relapse compared to those treated with placebo. This study…
Incyte has withdrawn its New Drug Application (NDA) for parsaclisib, which is an investigational treatment for various types of lymphoma, including relapsed/refractory mantle cell lymphoma (MCL), follicular lymphoma, and marginal…
A new Phase 2 clinical trial will begin this year to explore NKT2152 (a HIF2a-inhibitor) in combination with Fotivda/Tivozanib (a VEGFR tyrosine kinase inhibitor) as a treatment for relapsed/refractory clear…
Those with recessive dystrophic epidermolysis bullosa (RDEB) have fragile, easily blistered skin. Unfortunately, because of dysfunctional tissue repair, wounds may take longer to heal – causing increased levels of scarring…
Targeted Oncology recently featured a discussion by Dr. Jennifer Brown, director of Dana Farber’s Chronic Lymphocytic Leukemia Center, on the interim results from the SEQUOIA Phase 3 trial. The trial…
KalVista Pharmaceuticals plans to initiate a Phase 3 trial for KVD900, a treatment for hereditary angioedema (HAE) after finding positive results from earlier Phase 1 and 2 trials. The Phase…
According to a story from Al Jazeera, the country of Panama is the only one in Central America with a law on the books that is specifically related to rare…
Magnetic resonance imaging (MRI) has been used to study neuromyelitis optica spectrum disorders (NMOSD) in the past. Specifically, it is used to visualize the involvement of the spinal cord's central…
In the past, research has suggested that there was a genetic component to essential tremor (ET), a nervous system disorder. According to Medscape, researchers recently expanded on this understanding by…
Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…
Jordan's Guardian Angels, a nonprofit organization that supports the Jordan's syndrome community, has recently announced a new scholarship that will honor children that have died as a result of being…
It is common practice to treat multiple myeloma patients with a stem cell transplant. In the GENESIS trial, the stem cells were autologous (from the patient’s own peripheral blood or bone…
A Phase 3 clinical trial for advanced biliary tract cancer (BTC) has found that there is an improved overall survival rate when patients are given the immunotherapy durvalumab in combination…
In a late January 2022 news release, gene therapy company Taysha Gene Therapies, Inc. ("Taysha") shared that trial data was now available on TSHA-120. The therapy, designed to treat giant…
On January 27, 2022, The EveryLife Foundation for Rare Diseases, in partnership with the Biotechnology Innovation Organization (BIO), National Health Council, and Pharmaceutical Research and Manufacturers of America (PhRMA), hosted…
Fetal alcohol spectrum disorders (FASDs) occur following alcohol exposure during a mother’s pregnancy. But what is life like for those with fetal alcohol syndrome and their caregivers? The Irish Examiner…
AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…
PTC Therapeutics Inc. has just announced that their supplemental new drug application for Evrysdi (risdiplam) has been given priority review by the FDA. The aim is to expand the indication…
In a recent article for Hemophilia News Today, Jennifer Lynne, who is diagnosed with both von Willebrand disease and hemophilia B, stresses the importance of remembering women can have hemophilia…
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