According to a story from globenewswire.com, the biopharmaceutical company Liquidia Technologies, Inc. just announced that the company's New Drug Application (NDA) has been officially accepted by the US Food and…
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…
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…
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 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…
April is designated as Sarcoidosis Awareness Month. This year's event will be the sixth annual awareness campaign, which will involve collaboration between the Foundation for Sarcoidosis Research and the American…
According to a story from BioSpace, Acceleron Pharma Inc. and Bristol Myers Squibb have announced recently that the US Food and Drug Administration (FDA) has recently approved a new drug…
April 15th, 2020 will be the 7th annual International Pompe Day, a day of recognition and fostering awareness around Pompe disease organized by the International Pompe Association. Like many rare…
In this time of cancellations, closures, and shutdowns, many companies and individuals have found unique and creative ways to shift their services online. Better yet, some have temporarily waved their…
What is Repurposing? Repurposing involves testing whether or not a compound approved of another condition may be capable of also treating an illness with no approved treatment option. It's advantageous,…
According to a story from Vox, while the Center for Disease Control and Prevention (CDC) and other health authorities have reported the primary symptoms of coronavirus/COVID-19 to be a dry…
According to a story from The Columbus Dispatch, a variety of disorders, such as autism, can cause issues with sensory processing. What this means is that people on the autism…
According to a story from Sleep Review, the drug company Jazz Pharmaceuticals plc has recently filed a New Drug Application (NDA) for its investigational treatment JZP-258, which is being developed…
By now, everyone has heard of COVID-19. Information is constantly being updated surrounding the nature, symptoms, spread, and potential treatments for the illness. Despite this constant influx of information, there…
What is Homocystinuria? Classical homocystinuria, or HCU, is a rare metabolic condition which its caused by a deficiency of the CBS enzyme. It results in high levels of homocysteine in…
According to a story from CureDuchenne, the biotechnology company Dyne Therapeutics recently announced that CureDuchenne Ventures has made an equity investment that will support the development of all new precision…
On March 24th, 2020, Patient Worthy released a story announcing the formation of a Coronavirus Emergency fund by the American Kidney Fund (AKF). The goal of this fund is to…
According to a press release on Global Newswire, Novartis has received a positive opinion on Cosentyx® from the Committee for Medicinal Products for Human Use (CHMP). This positive opinion…
According to a press release from FirstWord Pharma, Gen and Alnylam Pharmaceuticals have signed an exclusive distribution deal in Turkey for RNAi therapeutic ONPATTRO. This therapy drug will treat…
According to a story from People's Pharmacy, US Surgeon General Jerome Adams recently stated in a tweet that regular citizens should stop buying face masks because they are not useful…
According to a story from Financial Buzz, the biotechnology company Inflazome recently announced that it has completed a phase 1 clinical trial. This trial was testing the company's experimental treatment…
Relapsing polychondritis is a rare disease which causes damage to the cartilage, trachea, and lungs. It's an autoimmune condition meaning an overzealous immune response causes the body to attack its own…
In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the Japanese Ministry of Health, Labour…
Published in Social Science & Medicine, a recent study conducted by Drexel University has shed light on how, even in a world of evolving precision medicine, there is also merit in…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
