According to a story from express.co.uk, multiple sclerosis (MS) is a neurological illness that can have major impacts on the lives of its patients. As a condition that can be…
If I told you that tiny bubbles could be used to more effectively treat cancer, would you believe me? Well, in the case of neuroblastoma, that might be correct. According…
Rare disease patients often face obstacles when it comes to the different aspects of care, whether that's diagnosis, screening, or management. Recent research from Baylor College of Medicine investigated the…
How many of you find yourself rushing around and it’s causing you stress and pain? And if you already have pain, does it add to your pain? Are you trying…
On July 28, 2021, biopharmaceutical company Thetis Pharmaceuticals ("Thetis") shared that its small molecule Resolvin E1 (RvE1) therapy, TP-317, received Orphan Drug designation from the FDA. While TP-317 has…
Updates from the Phase 3 clinical trial called FIGHT-302 were just released at the 2021 European Society for Medical Oncology World Congress on Gastrointestinal Cancer. The trial is comparing two…
In the United States alone, an estimated 300,000 people have moderate-to-severe chronic idiopathic urticaria (CIU), or chronic hives which are either recurrent or last for over 6 weeks, which…
Happy Friday! This week, we examine new findings that may reveal why cancer cells are able to resist an attack by immune cells. Also, why is a mother having to…
Orphan Drug designation is a status granted by the FDA to drugs or biologics intended to treat patients with rare conditions. In this case, the FDA defines "rare" as…
Previously, biopharmaceutical company XyloCor Therapeutics ("XyloCor") performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory…
According to a story from Market Watch, the biopharmaceutical company Ultragenyx Pharmaceutical, Inc. recently announced that it was given Orphan Drug designation from both the US Food and Drug Administration…
Prior to this year, there were no reported cases of monkeypox in the United States since 2003. This all changed when a Texas man brought the rare disease back with…
According to a relatively recent news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), interim data is now available from a Phase 1 clinical trial evaluating belcesiran for patients with…
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…
According to a July 27 news release from biopharmaceutical company Day One Biopharmaceuticals ("Day One"), the company's therapeutic option DAY101 received Rare Pediatric Disease designation within the United States. Altogether,…
In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…
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…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
A survey of oncologists has found that more education is clearly needed regarding cholangiocarcinoma. Specifically, more education would be beneficial regarding testing and targeting the mutations which cause the condition…
Bacterial meningitis is a severe condition that is linked to high mortality. Unfortunately, newborns are much more susceptible to bacterial meningitis. In fact, this infection occurs more than 30 times…
According to a story from MSN, mother Vanessa Ruiz is facing a terrifying choice: feed her family or pay for the genetic testing to confirm that her children Faith and…
Having a rare, chronic disease can impact one's life significantly, whether it's physically, spiritually, socially, or psychologically. Not only this, but those responsible for these patients' care are affected as…
Becca Meyers, a 26-year-old swimmer who was born deaf, has just been told she can't bring her care assistant to the Tokyo Games. As a result, she has had to…
On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…
The European Medicines Agency (EMA) recently received a Marketing Authorization Application (MAA) for Mycapssa, a maintenance therapy for acromegaly. Chiasma submitted the application, basing it on the results of their…
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