A recent article in Cancer Network announced that motixafortide met its primary endpoint in the GENESIS trial for multiple myeloma patients who were scheduled for stem cell transplantation. Often, the…
At birth, Don Smith was diagnosed with hemophilia A, a bleeding disorder. At that time, treatment options for the illness were limited. Most patients were not expected to live much…
Twins have always been a thing that has mystified the world, intrigued people, and definitely a curiosity. Fraternal, Identical, and everything in between – multiple births are a source of…
When is hearing loss a good thing to discover in a child? It sounds like the beginning of a “dad joke.” The answer and consequences are real, and in the…
In a sense, Langerhans cell histiocytosis (LCH) has always been somewhat of a mystery for doctors. As a rare multi-system disorder, LCH can affect the skin, lungs, bone marrow, spleen,…
Hyperphagia, or an abnormally stronger hunger or desire to eat, is one of the characteristics of Prader-Willi syndrome (PWS), a rare genetic disorder. Because of this, patients with PWS often…
Not many people expect a rare disease diagnosis out of the blue. For many, they had been seeking the correct diagnosis for years before finally being given the name of…
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…
Recently, there have been a number of reports of mucormycosis, a rare but sometimes fatal fungal infection, spreading throughout hospitals in India. According to MSN, the fungal illness is becoming…
According to a story from Renal and Neurology News, a recent study has revealed that lupus nephritis, a rare kidney disease associated with lupus, can suddenly recur even years after…
Just about one month ago, pharmaceutical company Secura Bio, Inc. ("Secura") shared that the company completed enrollment for the Phase 2 PRIMO clinical trial. Altogether, 101 patients enrolled. The…
Clinical trials can provide immense insight into potential treatment options and patient outcomes. Currently, biopharmaceutical company Protagonist Therapeutics, Inc. ("Protagonist") is working to gain insight into the efficacy of…
According to a recent article in Parkinson’s News Today, Dr. Per Borghammer, Research Professor at Denmark’s Aarhus University, proposed a new model that has the potential to identify early Parkinson’s…
Happy Friday! This week we have a moving patient story about how one mother's pregnancy led to an acromegaly diagnosis. Also, have you heard of Hereditary Tyrosinemia Type 1? If not,…
Becoming a new parent is always scary. There are plenty of worries and concerns: setting up a nursery, making the right decisions, and of course, the baby's health. Samantha and…
Receiving a rare disease diagnosis can be difficult, frustrating, and scary. Laura Hamilton, the host of A Place in the Sun, is no stranger to this phenomenon; last year she…
Written by Anis Mohd Nor I first became a mother in 2017. The pregnancy was a bumpy one - while I felt blessed to be pregnant after several years of…
On March 6, 2021, biopharmaceutical company Altavant Sciences ("Altavant") shared that it had initiated its Phase 2b ELEVATE 2 clinical trial. During the trial, researchers will evaluate rodatristat ethyl ("rodatristat")…
The month of May is recognized annually as CF (Cystic Fibrosis) Awareness Month, a time meant to help spread awareness about the genetic disorder among the medical community and the…
According to BioSpace, Farxiga (dapagliflozin), a treatment for patients with chronic kidney disease (CKD) and a risk of progression, recently became FDA-approved. Developed by AstraZeneca, the therapy is specifically designed…
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…
There are many reasons why medical research is important. It helps to amplify the patient voice, improve disease-related knowledge, and determine potential treatment goals. Additionally, medical research provides insight into…
Spinal muscular atrophy (SMA) is a rare, genetic disorder that impacts the muscles, making them progressively weaker over time. To combat this muscle atrophy, patients must exercise their muscles to…
In a recent study published in the scientific journal Cell Death & Disease, a novel proteasome inhibitor designated D395 was evaluated as a possible treatment for multiple myeloma, a rare form of…
On May 4th The International Waldenstrom's Macroglobulinemia Foundation (IWMF) held another webinar in its 2021 IWMF Global Education series titled Peripheral Neuropathy: No Easy Feat. As the title implies, the…
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