According to a story from the Irish Examiner, 31 year old Orla Tinsley is celebrating her first year of life following her life saving double lung transplant operation. This lung…
According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently announced that the first patient has been treated in the company's Phase 3 clinical trial testing sparsentan as…
According to a story from CBS News, Garland Benson, age 14, has undertaken a daunting task in order to save the life of his older sister Christiane. She was born…
Dr. Louis J. DeGennaro, who is the CEO and President of the Leukemia & Lymphoma Society, has recently contributed a blog post on the organization's website. In this post, Dr.…
According to a story from Science and Technology Research News, a group of researchers from the Cleveland Clinic led by Dr. Aziz Nazha will soon present a new myelodysplastic syndromes…
According to a story from Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…
According to a story from sectorpublishingintelligence.co.uk, the clinical stage pharmaceutical company Retrotope recently announced that the first patient had been dosed in its Phase 2/3 clinical trial. The trial will…
The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Patient Worthy. According to a story from Vox, a review of over…
According to a story from Scoop Independent News, citizens of New Zealand that have the rare bleeding disorder hemophilia A will soon have access to the drug emicizumab (marketed as…
According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…
According to a story from BioSpace, the biopharmaceutical company Merck recently announced that the US Food and Drug Administration (FDA) has approved the company's cancer drug KEYTRUDA in a new…
According to a story from WebMD, a recent study revealed that men with Crohn's disease, ulcerative colitis, or other forms of inflammatory bowel disease are at an increased risk of…
According to a story from gazettelive.co.uk, Jenni Mawson, who is currently 19 years old, has faced a years long struggle with chronic fatigue syndrome, which is also known as myalgic…
According to a story from BioSpace, a report on the results of the Orphan Drug Act of 1983 that was originally commissioned by the National Organization for Rare Disorders (NORD)…
According to a story from CBS News, Roxli Doss, an eleven year old girl, was diagnosed with a rare and dangerous brain tumor called diffuse intrinsic pontine glioma. It is…
According to a story from pm360online.com, the biotechnology company Amicus Therapeutics recently announced that the first patient has received treatment in the company's Phase 3 clinical trial testing the company's…
According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…
According to a story from thenewsreports.com, the biopharmaceutical company XORTX Therapeutics, Inc., recently announced that the company and its partner Cato Clinical Research have received correspondence from the US Food…
According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…
According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…
According to a story from the Translation Genomics Research Institute (TGen), the organization was recently awarded a grant of $10,000 from PayPal Gives. The funding will be allocated towards funding…
According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which…
According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…
According to a scientific study published in the Orphanet Journal of Rare Diseases, a team of researchers were able to identify the mutations responsible for choroideremia in five families of Polish descent.…
According to a story from freepressseries.co.uk, 45-year-old Stephen Millichamp was recently diagnosed with AL amyloidosis. The news was dire, and Stephen was told that the disease would ultimately be fatal.…
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