The 59th Annual European Society for Paediatric Endocrinology (ESPE) Meeting is taking place virtually from September 22-26, 2021. During the Meeting, researchers are discussing best practices and treatments, as well…
According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…
Treatment for patients with GM2 Gangliosidosis - which consists of Tay-Sachs disease and Sandhoff disease - has often been symptomatic and supportive. However, a new therapy shows promise in benefiting…
In the past, Exelixis' drug CABOMETYX (cabozantinib) has been approved for use in a variety of patients. For example, the treatment is approved for patients with renal cell carcinoma (RCC)…
According to a recent article published in The Online Trading Mentor, Inventiva S.A. (Inventiva) has recently opened enrollment for its Phase 3 trial of Lanifibranor, an investigational treatment for nonalcoholic…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or life-threatening illnesses, defined as those affecting under 200,000 people or…
In the United States, Fast Track designation is granted to help expedite drug development and review for serious, rare, or life-threatening conditions. To receive Fast Track designation, the treatment must…
Astellas Pharma has seen their ASPIRO trial put on clinical hold for the second time after the fourth patient passed away. The Phase I/II trial is evaluating AT132, a potential…
Endometrial cancer is the most common form of cancer that develops in the uterus, and there is currently a need for more treatment options, especially when it comes to the…
In the Phase 2 LOTIS-2 clinical trial, researchers evaluated the safety, efficacy, and tolerability of Loncastuximab tesirine for patients with aggressive diffuse large B-cell lymphoma (DLBCL). While there are treatment…
How does disease stage affect treatments and patient outcomes? In terms of adrenocortical carcinoma (ACC) in pediatric patients, disease stage significantly impacts patient outcomes. According to Cancer Therapy Advisor, the…
According to a news release from pharmaceutical company Citryll, the first healthy volunteer was dosed with CIT-013 in a Phase 1 clinical trial. Altogether, the drug is being developed to…
Recent data published in Gynecologic Oncology has confirmed the safety of olaparib for the treatment of advanced, BRCA-mutated, newly diagnosed ovarian cancer. This data came from the Phase 3 SOLO1 trial,…
Clinical trials can be beneficial for finding and evaluating new treatment options. Recently, biopharmaceutical company AstraZeneca shared how ALXN1840, a potential treatment for patients with Wilson disease, reached its primary…
For rare diseases in which no current treatment options are available, such as GM1 gangliosidosis, there is an urgent need to find therapeutics and improve patient outcomes. Gene therapy company…
Rare disease patients often face more obstacles and receive less aid than those impacted by more common conditions. There are many reasons for this inequity, but more research must be…
Have you ever wanted to be part of medical research? Well, now you might have the chance. According to Fox2Now, researchers from Saint Louis University - and, in particular, the…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare conditions, defined as affecting fewer than 200,000 Americans. As incentives, drug…
AstraZeneca is ending a phase 3 trial of Ultomiris, which was being evaluated as a treatment for amyotrophic lateral sclerosis (ALS). The pharmaceutical company acquired the C5 inhibitor after buying…
According to a recent press release from Pfizer and biotechnology company Vivet Therapeutics, VTX-801, a gene therapy candidate for patients with Wilson disease, recently received Fast Track designation from the…
Drug development is important in creating new therapeutic options for patients with rare or chronic illnesses. One incentivizing program is the Orphan Drug program. Orphan Drug designation is granted to…
According to a recent press release from biopharmaceutical company NFlection Therapeutics, Inc. (“NFlection”), the FDA granted Orphan Drug designation to the company’s drug candidate NFX-179. Altogether, this treatment is designed…
One key facet of the healthcare system is working to improve patient outcomes and finding new, effective, and innovative lines of treatment. According to Hematology Advisor, a study showed that…
Clinical trials are important tools in determining the safety, efficacy, and tolerability of new therapeutic options. So it is important to learn when patients are dosed, what those dosages are,…
In a recent news release, biopharmaceutical company Retrotope shared that enrollment was complete in its Phase 2 clinical trial. During the trial, researchers will evaluate RT001, a first-in-class oral…
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