According to a recent article in Parkinson’s News Today, the drug prasinezumab has entered the second half of a Phase 2 trial. An earlier trial, NCT02157714, showed that the therapy…
According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…
As originally reported in Biospace, Cushing disease, a disease due to excessive cortisol, has recently had its first ever treatment option approved by the FDA for adult patients who are…
Cuando algunas personas escuchan el "efecto placebo", evoca imágenes de píldoras de azúcar diseñadas para estimular la creencia del paciente. Pero según Healio, el término tiene un significado diferente…
According to a story from Biospace, in Palo Alto, AI company Emedgene plans to dive into helping the undiagnosed with their new rare disease initiative. The company will be paired…
COVID-19 and its rapid global spread have been anxiety-inducing, to say the least. For patients with rare diseases and their families, the growth of COVID-19 has also prompted additional…
According to a release on Biospace, the European Commission (EC) has approved Takeda Pharmaceutical's ALUNBRIG (brigatinib) as treatment for patients with anaplastic lymphoma kinase-positive (ALK+) advanced non-small cell lung cancer…
If you have read the news at all lately, you know that ventilators are a sought-after piece of medical equipment. With hospitals looking to treat mass amounts of patients…
Rare, neurologic diseases often do not have viable treatments. Gaucher disease and Parkinson's disease are two examples. In situations like these, medical professionals look to treatments for other conditions, such…
Shannon and Todd Palmatier have three children, two of which have progressive familial intrahepatic cholestasis (PFIC). Their sons' diagnoses have brought the lack of knowledge and advocacy to the attention…
Social Distancing Social distancing has been coined as the most surefire way to stop the spread of COVID-19 across the American populace. What is this phrase used to refer to?…
No one was given a prep course on how to handle quarantine, especially not with kids. Here are some suggestions to help you get through these unique times, while making…
As Patient Worthy readers, we hope you and your families are doing well during this challenging time. The health and safety of our rare community is always at the…
Results of one of the largest and most important studies in a decade, the International Study of Comparative Health Effectiveness with Medical and Invasive Approaches (ISCHEMIA), were just announced.…
The drug levodopa and most other therapies developed over the years for Parkinson's disease have been centered on correcting the lack of dopamine. Researchers acknowledge that the reason dopamine…
While many patients are grateful for their doctors and treatments, how many know why they are so passionate about what they do? In an interview with Dr. Patrick Flume,…
Hi everyone, Another week of life in COVID-19 era has passed! Today we have an article on a potential Coronavirus safety update. Next, we have the release of a new…
According to BioSpace, an experimental drug designed to treat cancer and HIV may also be poised to treat patients with COVID-19. The drug, called leronlimab, was created by biotechnology…
According to a story from People's Pharmacy, a dizzying array of advice related to minimizing spread or coronavirus/COVID-19 has barraged our senses over the last few weeks as the number…
A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…
As sourced from QZ, trials for new medications are notoriously tedious. Rare disease populations know more than most that the barriers to meeting the entry criteria of a trial can…
Every day on the news we hear the pleas for more medical masks for doctors and nurses, more personal protective equipment, and more ventilators. We all know what masks are,…
According to a study published in the journal Neurology, a measure called the vastus lateralis fat fraction was found to be useful in predicting the age at which a patient living…
Researchers at Columbia University have discovered a new gene, TBK1, that is linked to the progression of amyotrophic lateral sclerosis (ALS). A lack of understanding of a rare disease hinders…
According to a story from gurufocus.com, the biotechnology company Capricor Therapeutics has recently announced the initiation of a compassionate use program for its experimental drug CAP-1002. This drug will be…
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