Researchers have carried out a literature review to investigate MPS III. After reviewing forty-six papers, they concluded that more research needs to be carried out. You can find the original study…
A diagnosis of Cri du Chat (or 5P-), used to mean institutionalization. The name is french meaning "cry of a cat" which comes from the distinct sound children with the illness…
According to a report by medicalexpress, recent research from a DNA sequencing study revealed new information about Tourette syndrome. Led by researchers at UC San Francisco, this study represents the…
The United States Food and Drug Administration has approved Copiktra™ (duvelisib). The drug is now approved as a treatment for adult patients diagnosed with refractory or relapsed chronic lymphocytic leukaemia…
The company Genkyotex has announced that they have finished enrolling participants for a Phase 2 clinical trial of the investigational drug GKT831 in patients who have primary biliary cholangitis. The…
The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…
Encouraging interim data has been shared from two studies of risdiplam (RG7916), a drug being researched as a potential treatment for spinal muscular atrophy types 1, 2, and 3. For…
According to a story from MedCity News, a recent study revealed a potential weakness of CAR-T cell immunotherapies that reveals the need for precision and caution in the extraction process.…
How it all started Joy Milne is a retired nurse who helped diagnose her husband with Parkinson's disease. 12 years before the official diagnosis, Joy noticed a change in her…
A past, current, and future problem – underrepresentation in clinical trials, particularly for rare diseases. Why does representation matter? Besides, you know, the basics of equality, drugs can react differently…
October is Dysautonomia Awareness Month! Before we get into advocacy, let's touch on basic awareness... What is dysautonomia? Dysautonomia is the umbrella term that encompasses several different conditions that cause a…
Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old. Sutton is now 28 years old. Because of her condition, she must adhere to a very…
A Texas company, which opened in 2015, promised a new treatment for a number of conditions. Simply coming in and subscribing to a routine of IV treatments could treat cancer,…
According to a story from wtvbam.com, the drug development company Alnylam Pharmaceuticals has recently stated that it is looking less likely that its gene silencing therapy givosiran will be getting…
Just days after my CVID diagnosis, I was invited to a support dinner. It was at this support dinner that I became aware of how unique my situation was; I…
According to a story from McGill University, a group of Canadian researchers have discovered a mutation in the French-Canadian population that is linked to diseases including multiple system atrophy, Parkinson's…
According to a story from BioSpace, the biopharmaceutical company Orphazyme recently announced the results from its Phase 2/3 clinical trial of its investigational drug arimoclomol. This therapy is in development…
According to a story from BusinessWire, the drug developer Alexion Pharmaceuticals, Inc., recently announced positive results for its Phase 3 trial testing Soliris as a therapy for neuromyelitis optica spectrum…
The United States Food and Drug Administration has awarded Orphan Drug Designation to Q-Cells®, a product being developed by Q Therapeutics, for the treatment of transverse myelitis. For more detailed…
Avrobio has released updates on patients in two clinical trials of a potential gene therapy for Fabry disease. According to the announcement, two patients taking part in a Phase 1…
Ultragenyx Pharmaceuticals has shared data from two cohorts taking part in an on-going study involving an investigational gene therapy for ornithine transcarbamylase deficiency. For more information, you can view the…
My name is Lindsay Fogarty. I am a 25-year-old single mother to a wonderful 6, almost 7-year-old son, named Andrew. As I am writing this I am listening to the…
Topline results have been shared from a Phase 3 study of the investigational drug givosiran in patients with acute hepatic porphyria. For more detailed information you can view the source…
Many people express skepticism about the pharmaceutical industry. If not skepticism, than concern, if not wholesale distrust. For every few negative stories, however, there is at least one positive example.…
Many persons living with rare disease require a transplant of certain organs. Though this is not a cure for the rare disease Cystinosis, a kidney transplant is often required once the disease…
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