According to a November 3, 2021 news release from biopharmaceutical company OS Therapies, the company’s therapeutic candidate OST-HER2 (OST31-164 / Listeria monocytogenes) received Rare Pediatric Disease Designation from the FDA.…
In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…
It is often difficult to estimate the prevalence of a rare disease within the United States. For one, it can be difficult to find all of the patients with a…
According to a recent article, children with juvenile idiopathic arthritis experienced higher rates of joint inflammation during the COVID-19 lockdown. Juvenile Idiopathic Arthritis (JIA) Juvenile idiopathic arthritis (JIA) proves that…
LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…
Data from the TELEPRO-II study of Xermelo were recently published in the Journal of Cancer Management and Research. It investigated this oral therapy as a treatment for carcinoid syndrome (CS)…
In the European Union (EU), Orphan Drug designation ("Orphan designation") is granted to products intended to treat, prevent, or diagnose a life-threatening, chronic, or rare disease. These are defined as…
A recent article in NewScientist describes Borrelia burgdorferi as a bacterium that is the cause of Lyme disease (borreliosis). The trail of infection begins with wild mice that harbor the…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
A recent article in Science Alert reports on early but positive results from a first-in-human Phase 1 clinical trial of ProTide NUC-7738. About the Drug Researchers at Oxford University partnered…
While there is no cure for patients with systemic scleroderma, there are some therapies to manage symptoms, such as medications, antibiotics, physical therapy, or even surgery. But according to Medical…
Aurinia Pharmaceuticals Inc. plans to release the updated results from their continuation study of AURORA 2. This clinical trial is evaluating LUPKYNIS, also known as voclosporin, as a treatment for…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
According to a news article published by the Yale School of Medicine, Yale University researchers recently determined that autosomal dominant polycystic kidney disease (ADPKD) could potentially be reversed. Head researcher…
Currently, a glioblastoma multiforme diagnosis comes with a relatively poor prognosis: 25% survival after 1 year, and 5% after 5 years. Researchers have been looking to not only expand their…
Currently, there are no FDA-approved treatments for nonalcoholic fatty liver disease (NAFLD) or its more advanced version: nonalcoholic steatohepatitis (NASH). This is partly due to the lack of understanding surrounding…
Currently, about 90% of cystic fibrosis (CF) patients have a treatment option. This percentage jumped from 50% to 90% upon the approval of Trikafta. However, the remaining 10% of CF…
BRUKINSA (zanubrutinib), an orally administered Bruton's tyrosine kinase (BTK) inhibitor, has been approved in various countries for the treatment of mantle cell lymphoma (MCL), Waldenström's macroglobulinemia, and marginal zone lymphoma. For…
According to a news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the Osteogenesis Imperfecta Foundation (OIF), and the Osteogenesis Imperfecta Federation Europe (OIFE), enrollment is now complete for…
Currently, there are no known cures for CDKL5 deficiency disorder, a rare genetic disorder characterized by seizures and severe developmental delays. Treatment, rather, is symptomatic and supportive. However, a news…
In the past, some researchers believed that fibromuscular dysplasia was a rare disease. While some researchers estimate that the incidence is actually much higher than reported, there is still a…
Atara Therapeutics has released data from its Phase 1 open-label extension (OLE) trial of ATA188, a treatment for progressive multiple sclerosis (MS). This allogeneic T-cell immunotherapy targets B cells and…
Silica is a naturally occurring substance found in various types of rocks, clay, and sand. Unfortunately, when this silica dust is inhaled, especially over long periods of time, it can…
Written by Lori Lawter, MPH We’ve heard a lot about bringing drugs to market lately, especially in light of the COVID-19 vaccine. You might wonder, “Is there a ‘secret sauce’…
For many patients, symptoms of Huntington’s disease appear within midlife: between the 30s and 40s. This progressive disorder causes brain cell death, leading to a loss of physical and mental…
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