Hannah Creel was only 18 months old when she was first diagnosed with cystinosis, a rare disorder that affects all organs in the body, in particular the kidneys. Her parents…
When Taylor Brown was just two years old, her mother TaMetress Reed began noticing certain symptoms, such as hardening and tightening patches of skin. As Brown grew up, the symptoms…
According to a relatively recent news release from biopharmaceutical company Applied Therapeutics, Inc., AT-007 received Fast Track designation from the FDA. This therapy, which has also received both Orphan Drug…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
We're still trying to understand COVID-19, and it may take us years and years to fully comprehend the respiratory virus. A recent case study published in the Canadian Medical Association…
Internationally, over 400,000 people die of malaria each year. In many cases, a majority of these deaths occur in sub-Saharan Africa; children are the most vulnerable. However, according to Medical…
You may know Jessie J from some of her hit singles like "Price Tag" or "Bang Bang," but did you know that she is also a rare disease patient? She…
For the first time, a research study has discovered a genetic marker related to high-risk neuroblastoma. According to Medical XPress, researchers determined that anaplastic lymphoma kinase (ALK) gene mutations and…
As reported by Oncology Nursing News, gunagratinib (ICP-192) was recently granted Orphan Drug designation by the FDA. Altogether, this treatment is being evaluated as a potential treatment for patients…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
In an innovative collaboration, the TAPS Support Foundation and the Fetal Therapy team from the Leiden University Medical Center announced today a new joint initiative – The Twin Talks Webinar…
The pressure was on. Researchers had been studying Alzheimer’s for decades. Every morsel of discovery was hailed as moving closer to a cure and results were always expected soon…
Currently, there are no real treatments for patients with primary mitochondrial myopathy (PMM). Most therapies, such as physical, occupational, or speech therapy, are designed to simply lessen or mitigate symptoms.…
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…
In late May 2021, the FDA granted accelerated approval to Truseltiq (infigratinib). As reported by Cure, this therapy is designed for adult patients with unresectable locally advanced, previously treated, or…
A recent study presented in early June showcased how, despite some similarities, psoriatic arthritis with axial involvement (axial PsA) patients and axial spondyloarthritis (axSpA) patients have meaningful differences. These findings…
According to a recent article in Biospace, patients with advanced systemic mastocytosis (SM) can turn to a newly FDA-approved treatment by Blueprint Medicines called Ayvakit (avapritinib). The drug is the…
University of Illinois Researchers have uncovered novel information about pulmonary fibrosis. Their research demonstrates that fibrosis in the lungs can be reversed, and tissue can be repaired using microgel-coated mesenchymal…
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A recent study aimed to uncover the incidence of osteomyelitis in Germany. The researchers explain that because the epidemiology of the disease is unknown, it is hard to estimate the…
Rare disease patients often face more obstacles than others when it comes to healthcare. While we know this, it can be hard to judge the public's stance on the issue.…
When Stephanie Ernst-Milner first discovered that she was having twins, she was speechless. But what she learned over the course of her pregnancy, and in the seven years since, has…
The Quincy community is helping to organize a bone marrow registry drive for a local teen who was recently diagnosed with hemophagocytic lymphohistiocytosis (HLH), according to an article in The…
As reported in PR Newswire, Alcyone Therapeutics is launching a new set of precise gene therapies for CNS disorders with the help of a new $23 million investment from RTW…
States are not able to alter patent protections or set prices but Colorado is no longer waiting for the federal government to act. According to a recent article in…
WLNS Brooklyn Michigan is sharing news about Jaxon Meschke, a four-year-old boy with a rare disease called cystinosis. Jaxon’s body has been fighting the disease since he was six…
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