In April 2022, the United States approved Ultomiris for the treatment of patients with generalized myasthenia gravis (gMG). This approval was followed, just four months later, by an approval…
The new nonprofit Every Cure has been created on the basis that every drug currently on the market effects multiple areas in the body. This premise has been proven…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…
According to an article from Cancer Network, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to second-line GNS561 (ezurpimtrostrat) for cholangiocarcinoma (bile duct cancer). In…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from neurology.duke.edu, Duke University has recently been recognized as a Center of Excellence for Dystonia treatment by the Tyler's Hope for a Dystonia Cure Foundation. This…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs designed for patients with rare or life-threatening conditions. Now, the FDA grants…
ScienceAlert recently published an article explaining how scientists at Rice University have been able to eradicate tumors in mice by using a new treatment that takes less than a…
According to a story published in Healio, a recent study has concluded that a combination of standard treatment and belimumab produces better outcomes for patients of East Asian descent living with…
According to an article from Yahoo! Finance, biopharmaceutical company Belite Bio, Inc. has initiated the Phase 3 DRAGON clinical study to evaluate LBS-008 for individuals with Stargardt disease. The…
Human papillomavirus (HPV) types 16 and 18 play a huge role in the development of cervical cancer. In fact, an estimated 70% of cervical cancer cases result from these…
Currently, there are an estimated 30 million people within the European Union (EU) who have a rare disease. The “Orphan Medicines” (or Orphan Drug) program was designed to facilitate…
David Guy, parent of Kaila who was diagnosed with acid sphingomyelinase deficiency (ASMD), recounts his initial shock when he was told of Kaila's rare disease. David and his wife…
When we ‘do the math’ it adds up to the novel AMT-130 gene therapy receiving positive results from uniQure’s clinical trial investigating a small group (N=4) of individuals who were…
Dr. Jung-Mo Ahn, associate professor at Dallas University in Texas, created a molecule that kills a broad range of treatment-resistant cancers. The newly targeted group includes triple-negative breast cancer.…
On August 25th, 2022, the American Society of Gene & Cell Therapy held a webinar as part of its Lunch and Learn series, which is intended to help people living…
Unfortunately, the drug development process can be difficult, especially in the rare disease sphere. Many drug developers choose to focus on larger disease states, as these are typically more…
In June 2022, the FDA approved Amvuttra, a prescription medicine designed to treat polyneuropathy (nerve damage) due to hereditary transthyretin amyloidosis (hATTR amyloidosis). This approval was huge. Typically, managing…
Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…
By definition, NASH or nonalcoholic steatohepatitis is a form of fatty liver disease. NASH may be a result of a metabolic disorder such as diabetes or obesity that develops…
According to a story from Benzinga, the pharmaceutical company Inceptua Group has recently announced that the medicine paclitaxel micellar (marketed as Apealea) is now available in Germany. This medication is…
According to a story from Market Screener, the biotech company UNITY Biotechnology, Inc., has recently announced the release of data from 12 weeks and 18 weeks of its Phase 2…
According to a story from Upstate Medical University, the college's ALS Treatment and Research Center has been a certified center of excellence for research and clinical care that has helped…
According to a story from Kalimpong News, over 300 children with rare diseases in India are still waiting for the release of pledged funds from the Union Health Ministry so…
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