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 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…
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…
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…
An art exhibit held at the Galerie Gora in Montreal, Canada, has used photographs and short stories to share the experiences of eleven people with acromegaly. You can read the…
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…
Shriners Hospitals for Children® has announced their motion analysis centre consortium. The hospital’s Motion Analysis Centre is used to evaluate the movements and gait of children and teenagers. To read about…
Nightstar Therapeutics has released preliminary data from a Phase 1/2 study of an experimental gene therapy designed to treat X-linked retinitis pigmentosa. For more detailed information you can view the…
A study carried out by researchers from Harvard Medical School and Brigham and Women’s Hospital has explored the link between dopamine neurones, Parkinson’s disease, and psychiatric disorders. To read about…
The United States Food and Drug Administration has awarded Orphan Drug Designation to an experimental PLX cell therapy (called PLX-R18) as a treatment for graft failure and incomplete haematopoietic recovery…
Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…
A study carried out by a research team at Yale University and mainly funded by the National Institutes of Health has identified a cell in the spinal fluid of patients…
After their own personal struggles with cancer, the Rendina family are working to raise money and awareness to fight the disease. For more information, you can view the source press…
Researchers at the University of Pennsylvania have studied the genetics of a class of neurological disorders that includes Huntington’s disease and Fragile X Syndrome. They found that there were similarities…
Maurice Elias, who was born with severe combined immunodeficiency, has met up with the doctor who saved his life almost fifty years ago. For more information, you can read the…
Scientists working in a lab have successfully managed to grow a miniature human esophagus (esophageal organoid). They hope that this could be used in the future to study conditions…
A new study explored the mechanisms underlying narcolepsy, a rare sleep disorder. The condition has previously been linked to reduced levels of a neurotransmitter called hypocretin, and the recent study…
A new study has found that a certain type of bacteria may play a significant role in the development of stomach cancer. For more information, you can view the source…
A research team led by scientists from Newcastle University believe that they may have identified a previously unknown genetic cause of childhood mitochondrial disease. For more information about this, you…
Amicus Therapeutics has met with the United States Food and Drug Administration to discuss the investigational drug AT-GAA, which is being researched as a potential treatment for Pompe disease.…
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