In a recent press release, RNA-targeted therapeutics company Ionis Pharmaceuticals, Inc. ("Ionis") shared the initiation of a Phase 2/3 clinical trial evaluating ION373 for patients with Alexander disease (AxD). Currently,…
Normally, metformin is used to help control blood sugar in patients with type 2 diabetes. However, new research suggests that the treatment option could also be beneficial for those with…
A variety of gene mutations cause retinitis pigmentosa (RP) - but how do those genes affect the degeneration of rod and cone photoreceptors in the eyes? According to Medical XPress,…
As many patients within the rare disease community know, the diagnostic odyssey can be long and arduous. But what if burgeoning technologies, like artificial intelligence (AI) and its branches (machine…
During clinical trials, Independent Data Safety Monitoring Boards (DSMBs) sometimes assess trial safety, trial integrity, and how well the trials are upholding patient needs. According to Business Insider, the DSMB…
With genetic disorders, there is always a big question: do specific gene mutations respond differently to treatment? How can treatment be targeted to best suit the needs of patients? According…
Did you know that zebrafish are often used in genetic research? Not only do they have the same major organs and tissues as humans do, but zebrafish also share a…
Every year, an estimated 3-7 children in Norway are born with phenylketonuria (PKU), a rare genetic disorder characterized by an inability to break down phenylalanine. Researchers in Norway wanted to…
Each year, researchers make advances in the medical field which assist with the identification, diagnosis, and treatment of patients with rare conditions. According to Genetic Engineering & Biotechnology News, one…
In a recent press release, clinical stage biotech and biopharmaceutical company Biom Therapeutics ("Biom") shared that its drug candidate BIO017 was granted Orphan Drug designation. The treatment, designed for patients…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
Despite the pandemic, there were many strides in the field of medicine and research in 2020. For example, researchers first discovered and identified a rare disease called VEXAS syndrome. The…
Fast track designation is granted by the FDA. If a drug developer receives this designation, they begin a process to develop and expedite review for drugs or biologics which treat…
Is it possible for certain gene mutations, which cause a specific condition, to also raise the risk of developing another condition? Well, says HealthDay, the answer might be yes. In a…
Altogether, there are over 7,000 rare diseases. Worldwide, 1 in 20 people live with a rare disease. But the journey to diagnosis can sometimes be confusing or difficult. So when…
For more than 30 years, Ionis Pharmaceuticals, Inc. ("Ionis") has worked to create RNA-targeted therapies for patients in need. Now, according to a recent press release, the company initiated…
In a press release from March 3, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared that it had completed full enrollment for a Phase 2 clinical trial evaluating vosoritide for…
At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
During the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy, specialty pharmaceutical company Italfarmaco Group ("Italfarmaco") shared updates regarding various Givinostat clinical development programs. The treatment is designed…
February 28th is known as Rare Disease Day, designed to raise awareness of rare diseases, patients, caregivers, and their experiences. As this day approaches, the American Kidney Fund (AKF) shared…
New research suggests that ultrasounds could be a new diagnostic tool for patients with a rare lysosomal storage disorder. According to Fabry Disease News, kidney dysfunction is characteristic in Fabry…
Just over a week ago, drug discovery and development company Rescindo Therapeutics Inc. ("Rescindo") shared that its drug candidate RSC-57 received both Orphan Drug and Rare Pediatric Disease designations. The…
According to Epidermolysis Bullosa News, staph infections are increasingly common in the wounds or blisters of patients with epidermolysis bullosa (EB). In fact, an analysis of these lesions showed that most had…
According to ForexTV, commercial gene therapy company Spark Therapeutics ("Spark") recently announced preliminary data from a Phase 1/2 clinical trial evaluating SPK-8016, an investigational gene therapy, for patients with hemophilia…
In a recent press release, biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum") shared that the company finished its rolling New Drug Application (NDA) submission. The NDA focuses around maralixibat, an investigational…
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