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Incyte has withdrawn its New Drug Application (NDA) for parsaclisib, which is an investigational treatment for various types of lymphoma, including relapsed/refractory mantle cell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…
Currently, there are a number of treatment options for patients with follicular lymphoma. But for patients whose condition has relapsed despite prior treatments, there are very limited therapeutic options. After…
Jaguar Health and Napo EU S.p.A have just announced that crofelemer, an investigative therapy for short bowel syndrome (SBS) has been granted Orphan Drug Designation by the EMA. Thanks to…
Diagnosing a rare disease early is important, as it allows for prompt intervention and treatment. Recently, researchers took large steps forward in the diagnostics of pulmonary arterial hypertension (PAH) in…
Rare disease patients know what it's like to go without a diagnosis despite enduring symptoms. Doctors often don't recognize the disorders for a number of reasons, whether that's the similarity…
A recent study published in Advances in Rheumatology has uncovered 2 potential biomarkers which could be used to predict which lupus nephritis (LN) patients are at the highest risk of severe renal…
Researchers from the Perelman School of Medicine at the University of Pennsylvania recently explored the underlying biological cause and mechanisms behind fibrodysplasia ossificans progressiva (FOP). In the past, researchers from…
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