According to a recent article, patients diagnosed with a rare disease are faced with incredibly high health care costs compared to those without a rare disease. Impact of Rare Disease…
When it comes to advances in hereditary ATTR amyloidosis (hATTR amyloidosis), biopharmaceutical company Alnylam Pharmaceuticals, Inc. ("Alnylam") has been on top of its game. Alnylam helped develop Onpattro and is…
On October 30-31, 2021, the Periodic Paralysis Association (PPA) hosted the Periodic Paralysis Conference in Orlando, FL. This event was an opportunity for patients to meet one another, share experiences,…
LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…
Data from the TELEPRO-II study of Xermelo were recently published in the Journal of Cancer Management and Research. It investigated this oral therapy as a treatment for carcinoid syndrome (CS)…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
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 recent article from MedPage Today, new clinical guidelines involving parathyroidism were previewed at the American Society for Bone and Mineral Research (ASBMR) meeting last month. Hypoparathyroidism Hypoparathyroidism…
Like every other body part, bones are living tissues and go through several rejuvenation cycles throughout life. Older bone tissues are replaced with newly formed bone tissues in a process…
Albert Edge and Yushi Hayashi recently led a research team which uncovered the specific mechanism which can cause deafness in Norrie disease, a rare condition. These findings were recently published…
On October 27, 2021, the National Organization for Rare Disorders (NORD) hosted an informational webinar titled "Navigating Diversity, Equity, and Inclusion (DEI) in Rare Disease Non-Profits." This webinar was the…
Access Opportunities for Rare Disease Patients At present, there are around 7,000 identified rare diseases, with only 5% having treatments. That leaves more than 95% of rare disease patients with…
According to a press release from Ikena Oncology, the FDA has recently accepted an Investigational New Drug (IND) application for Ikena's investigational drug candidate, IK-930. This TEAD inhibitor is intended…
Written by Rod Cisneros Like many people, I keep a daily journal. I use it to document my everyday thoughts and I even include advice for family members who might…
In the European Union (EU), Orphan Drug designation ("Orphan designation") is granted to products intended to treat, prevent, or diagnose a life-threatening, chronic, or rare disease. These are defined as…
A recent article in NewScientist describes Borrelia burgdorferi as a bacterium that is the cause of Lyme disease (borreliosis). The trail of infection begins with wild mice that harbor the…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
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…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
A recent article in Science Alert reports on early but positive results from a first-in-human Phase 1 clinical trial of ProTide NUC-7738. About the Drug Researchers at Oxford University partnered…
The University of Pittsburgh School of Medicine along with Prairie View A&M University, have recently uncovered that there are some cancer drugs which could be used as an effective treatment…
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 a…
An October 2021 report published in the Globe and Newswire carried an announcement by Fabio Chianelli, CEO of the biotech company PharmaTher Holdings. The FDA approved orphan drug status for…
Aurion Biotech has just released the results from their clinical trial called IOTA. This trial was conducted by researchers in Japan and demonstrated that corneal endothelial cell therapy was able…
Cooking and eating together is a great way to build closer bonds and spend time with those we love. In Malaysia, it's a large part of the culture, as there…
© 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.
