LiPlaCis is a formulation of cisplatin that delivers its agent directly to cancerous tumor sites. Its efficacy has so far only been studied within breast cancer patients. However, scientists have reason to…
A drug called Coversin is being investigated as a potential treatment for four orphan diseases: bullous pemphigoid (BP), atypical keratoconjunctivitis (AKC), thrombotic microangiopathies, and paroxysmal nocturnal hemoglobinuria (PNH). Recently, new…
PharmAbcine has announced that the United States Food and Drug Administration has responded to the Investigational New Drug application they submitted on behalf of an experimental drug called TTAC-0001, which…
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…
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 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…
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…
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…
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…
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.…
According to a story from Parkinson's News Today, a recent analysis of Parkinson's disease research showed that scientists would benefit from relying less on animal models and instead should focus…
Two Phase 2 clinical trials called ‘Elektra’ and ‘Arcade’ have been initiated. They will investigate the drug OV935/TAK-935 as a potential treatment for paediatric patients with rare epilepsies. For more…
Researchers at the University of Utah are working on developing computational tools that can be used to help identify the genetic changes underlying early infantile epileptic encephalopathy. For more detailed…
Avrobio has received no objection from Health Canada for their clinical trial application for their Phase 1/2 study of an investigational gene therapy for Gaucher disease. Following this news, Avrobio…
According to a story from news-medical.net, a genomics study called Angiosarcoma Project has successfully discovered that the inhibition of immune checkpoints could have therapeutic value for patients with angiosarcomas affecting…
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