The FDA has a series of designations it grants to drugs or biologics intending to treat rare, serious, or life-threatening conditions. One of these is Breakthrough Therapy designation. According to…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Happy Friday! This week, author Tom Seaman asks: When it comes to anxiety, are you turning your Gizmo into a Gremlin? Also, in our new Compassion Corner series, we examine…
Unfortunately, clinical trials do not always go the way that researchers intend. Sometimes, issues arise during early or preclinical studies which cause enough concern to stop or pause the…
According to MPR, the FDA recently granted Orphan Drug designation to CPI-613 (devimistat), an investigational treatment designed for patients with biliary cancer. Altogether, "biliary cancer" can consist of bile…
In the past, some drug developers were not incentivized to develop treatments for those within the rare disease community, considering the smaller patient size. However, the FDA created the Orphan…
We know that it can be difficult to receive the correct diagnosis for a rare disease. Sometimes, you have to be your own advocate. This means learning everything you can,…
Want to surgically remove a pancreatic tumor? Well, the entire process can take anywhere from 6-12 hours and presents some difficulties for medical practitioners. For example, during surgery, it can…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
Amyotrophic lateral sclerosis (ALS) is a rare, neurological disorder that is characterized by the progressive death of nerve cells and muscle weakness. As time passes, many affected individuals experience difficulty…
St. Jude Children's Research Hospital has recently announced that its team of scientists have established a registry for pediatric melanoma. The goal is to use data from this registry for…
My nickname is Arti and I'm 11 years old. Because of my HAE diagnosis, I've been suffering from frequent and painful swellings since I was little. My life is divided…
Rare diseases face many obstacles when it comes to research and drug development. Small patient populations, a lack of awareness, and limited funding are just a few examples. When it…
The month of July is recognized as Sarcoma Awareness Month, a time of the year for spreading awareness about sarcoma among the general public and in the medical community. An…
Often misdiagnosed as other conditions such as Rett syndrome or cerebral palsy, DDX3X mutations cause both cognitive and physical difficulties. Since only an estimated 700 patients have been diagnosed…
In the past, arimoclomol has not always shown treatment promise. For example, the drug failed to meet primary or secondary endpoints in a clinical trial evaluating arimoclomol for patients with…
According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…
Unfortunately, many molecules which drive cancer development and growth are considered "undruggable." In short, this means that drugs are unable to bind to or address these molecules due to size,…
Even though my boyfriend's mom works at an eye doctor, I'm still kind of afraid to get eye exams - and I'm not alone. Unfortunately, most people do not receive frequent enough…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The European Hematology Association (EHA) held its 2021 Virtual Congress from June 9-17, 2021, though the insights will be available until mid-August 2021. During the Congress, researchers discussed new…
A new study, funded by the NIH and conducted by Vanderbilt University Medical Center (VUMC), is working to assess allergic reactions to COVID-19 vaccines. The trial will include individuals between…
Two new reports of Guillain-Barré syndrome (GBS) have been documented in response to the AstraZeneca COVID-19 vaccine. Previously, published papers have documented 4 cases in England and 7 cases in…
Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has…
© 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.
