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.
The US FDA has awarded Orphan Drug Designation to the experimental drug CPI-613, for the treatment of Burkitt lymphoma. The full article can be read here, at Globe Newswire. About…
The Australian government will give $33 million to researchers to study rare cancers and rare diseases. The original article can be read here, at the Department of Health’s website. Background…
Results from a Phase 3 trial of the medicine Lynparza (olaparib) for the treatment of advanced ovarian cancer in women with the BRCA-mutation have been announced as positive. The full…
Amicus Therapeutics is developing a treatment for Pompe disease through its AT-GAA program. They have recently announced that they have met with regulators from the European Union and Germany who…
This article discusses the plot of the biographical film Brain on Fire. Susannah Cahalan was twenty-four when she began experiencing seizures, hallucinations, and, after only a few weeks, catatonia. Her…
The drug Jakavi (ruxolitinib) is being investigated as a possible treatment for polycythemia vera (PV) and myelofibrosis. Results from studies into this were presented by Novartis at the 23rd Congress of…
A study that looked into what genetic factors influence medulloblastoma development has been published in Lancet Oncology. In it, researchers outline several genes they believe are linked to tumour formation…
Avedro has announced that patients are now being enrolled in a Phase III trial of an epithelium-on (epi-on) corneal collagen cross-linking procedure designed as a treatment for patients who have…
Arrowhead Pharmaceuticals, Inc. have announced that they have completed patient enrollment for a Phase 1 clinical trial investigating ARO-AAT, an investigational therapy for the treatment of a genetic liver disease…
A Phase I/II clinical trial of the investigational gene therapy SGT-001 designed to treat Duchenne muscular dystrophy has had its clinical hold lifted by the US Food and Drug Administration…
Prana Biotechnology has announced that it has begun recruiting healthy volunteers for its Phase I clinical trial of the experimental drug PBT434. The drug is thought to affect processes linked…
Data from a Phase II clinical trial of the experimental treatment reldesemtiv for the treatment of spinal muscular atrophy has been announced. The drug has shown positive effects in the…
An experimental gene therapy designed to treat Duchenne muscular dystrophy has produced exciting preliminary results in a Phase 1/2a clinical trial. Sarepta Therapeutics, the company behind the therapy’s development, announced…
An open-label extension of a Phase II study has shown that the experimental drug lenabasum is fairly safe and tolerable in patients who have diffuse cutaneous systemic sclerosis. The full…
The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…
Clinical data from a Phase 2 study of the experimental drug X4P-001-RD for the treatment of WHIM syndrome has been announced at the European Hematology Association’s 23rdCongress in Stockholm. The…
The first patient has undergone stem cell transplant treatment in the third cohort of a Phase I clinical study of the experimental treatment ISC-hpNSC for Parkinson’s disease. The full article can…
The first patients have been enrolled in two clinical trials of the experimental drug neridronate. The trials are part of the company’s Phase 3 program to evaluate the effects of…
The U.S. Food and Drug Administration (FDA) has approved the medicine Keytruda for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma, that have relapsed…
Researchers have discovered a new drug that appears to slow cell growth in mantle cell lymphoma. The full article can be read here, at the Bellvitge Biomedical Research Institute (IDIBELL)…
The All of Us program is a huge research project set up by the National Institute of Health (NIH) that aims to collect genetic and health data from a representative…
A phase 1/2 study of the drug lumasiran for the treatment of primary hyperoxaluria Type 1 has produced encouraging results, reports Alnylam Pharmaceuticals. The full article can be read here,…
Promising data from the ongoing Phase I clinical trial Explorer has been announced by Blueprint Medicines Corporation. The trial is designed to evaluate the effects of the experimental drug avapritinib…
The U.S. Food and Drug Administration has granted the experimental drug A4250 rare paediatric disease designation for the treatment of progressive familial intrahepatic cholestasis. The full article can be found…
Lawmakers in the U.S. state of North Carolina have expanded the state’s newborn screening testing to include three more conditions. These are Pompe disease, Mucopolysaccharidosis type 1 (MPS 1), and…
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