Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.
A recent article published by the University of California San Francisco looks back at the development of gene therapy technology and explores how scientists at the university are working to…
RAREfest, an event organised by the Cambridge Rare Disease Network (CRDN), will bring together patients, scientists, researchers, clinicians, support groups, businesses, and the public, to engage and educate people about…
Goethe University in Frankfurt, Germany has been awarded a grant of more than one million euros as part of a large-scale project in Europe to standardise and make available clinical…
An on-going study of an investigational drug for primary biliary cholangitis has been given the go-ahead to continue by a safety monitoring board. The planned review recommended that the Phase…
The Cambridge Rare Disease Network’s RAREfest, a festival focusing on rare diseases, will bring together researchers, scientists, patients, and the public. It’s taking place on the 30th November and 1st December in…
Novartis has announced positive findings from their research into brolucizumab, an investigational drug being developed to treat neovascular age-related macular degeneration. This data comes from year two of the…
Results from a study that were presented at the European Society for Medical Oncology’s Congress have shown that the investigational drug for prostate cancer INO-5150 may be able to slow…
A new study into a potential drug for cystic fibrosis has begun. The Phase Ib/IIa trial will investigate the drug POL6014 in up to forty patients who have cystic…
A recent article titled ‘How I use molecular genetic tests to evaluate patients who have or may have myelodysplastic syndromes’ published in the journal Blood discusses the role of genetic testing for…
An experimental therapy for melanoma patients has shown anti-tumour activity in an ongoing Phase 1 study. The therapy, called pegzilarginase (AEB1102) is being developed by Aeglea BioTherapeutics. For more…
Loxo Oncology has shared interim clinical data from an ongoing study of the drug LOXO-292. The study, a Phase 1/2 clinical trial, involves patients with RET-mutant medullary thyroid cancer, as…
A major genetic research project called the Deciphering Developmental Disorders (DDD) study is helping families to receive diagnoses, and, in some cases, even identifying new disorders. For more information,…
In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…
New data has been released about the effects of lenabasum, an experimental drug, on patients with systemic sclerosis and dermatomyositis. Corbus, who are developing lenabasum, are planning to present the…
Last time Patient Worthy wrote about Mila Makovec, she was seven and beginning a cutting-edge experimental treatment for her Batten disease. It’s now nine months later, and Mila is showing…
Multimorbidity, typically defined as having two or more long-term health conditions, has become a topic of increasing focus for healthcare providers. Many elderly people are affected by multimorbidity, with the…
An ambitious project called the Human Cell Atlas is attempting to study all the cells in the human body – of which there are more than 37 trillion. Researchers hope…
According to a recent press release by Anavex, the United States Food and Drug Administration has approved an investigational new drug application for a study of the drug Anavex®2-73 in…
Genomic testing is becoming increasingly common, but more work still needs to be done into understanding which gene variants are linked to health and disease. To address this, the National Institutes…
The companies Genentech and Roche are working together to carry out a program of studies involving patients with Huntington’s disease (the huntingtin-lowering trial ASO program). One is a natural history…
The United States Food and Drug Administration has granted the drug Lynparza® (olaparib) Orphan Drug Designation for the treatment of pancreatic cancer. Lynparza is used in the US to treat…
The United States Food and Drug Administration has granted Fast Track designation to a drug being researched as a potential treatment for progressive familial intrahepatic cholestasis, a serious liver disease…
The United States Food and Drug Administration has awarded Rare Pediatric Disease Designation to a potential drug for Mucopolysaccharidosis type I. For more detailed information about this news, you can…
The United States Food and Drug Administration has awarded Orphan Drug Designation to an investigational drug for narcolepsy, and there are plans to carry out a Phase 2 study to…
Encouraging topline results from a pivotal Phase 3 study into a drug for Charcot-Marie-Tooth type 1A disease have been shared by Pharnext. To find out about this news in more…
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