According to a story from Financial Buzz, the biopharmaceutical company Mereo BioPharma Group plc recently announced that it has successfully concluded its Type B End-of-Phase 2 meeting with the US…
In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…
According to a story from mdmag.com, a recent proof-of-concept style study suggests that a certain machine learning tool could be of benefit to patients living with the rare condition alkaptonuria.…
According to a story from Sixth Tone, life for the Lu family of Yuncheng, China, has never been easy. Life in the region has slowed thanks to the coronavirus, but…
This year, the entire Patient Worthy team traveled up to Washington, DC for Rare Disease Week 2020. This event is always a big one for the rare disease patient community.…
The Jack McGovern Coats' Disease Foundation, a nonprofit patient organization committed to supporting critical disease research and raising disease awareness, is offering travel grants for recipients to attend the 2020…
According to a story from PR Newswire, scientists are making significant progress in researching new treatments for glioblastoma, a rare and deadly brain cancer. A variety of drug companies are…
One of the less well known forms of primary immunodeficiency is called activated PI3K delta syndrome (APDS). It is a rare disease that has only recently been identified by scientists.…
According to a story from Financial Buzz, the ophthalmology company Acucela Inc. has recently announced an update on the progress of its phase 3 clinical trial. This clinical trial is…
According to a story from stcatherinesstandard.ca, former firefighter Tom Salisbury was 54 years old when he was first diagnosed with a form of vasculitis, a type of rare autoimmune disease.…
According to a story from Healio, Cushing's disease, a cause of Cushing's syndrome, can still be a deadly disease if it remains untreated. However, recent developments in the treatment of…
According to a story from Newsfile, the biotechnology company PharmaCyte is beginning to turn heads with the potential of its proprietary Cell-in-a-Box technology that has the potential to greatly improve…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
According to a story from gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…
According to a story from Benzinga, the biotherapeutics company CSL Behring has recently announced that its medical product PRIVIGEN ® has earned Orphan Drug designation from the US Food and…
According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held in Orlando, FL. These updates…
According to a story from Targeted Oncology, findings from a recent phase 1 trial have discovered a new potential treatment for myelofibrosis. The study combined two different drugs: panobinostat (marketed…
According to a story from gurufocus.com, the pharmaceutical company Zogenix, Inc., has recently announced top-line results from its phase 3 clinical trial. This trial is testing FINTEPLA® as a treatment…
According to a story from Targeted Oncology, a recent phase 1/2a clinical trial of an experimental treatment called TAK-007 produced a clinical response in the majority of patients in the…
According to a story from docwirenews.com, health-related quality of life (HRQOL) is going to be significantly impacted in patients with chronic kidney disease (CKD), especially as the disease progresses. Biomarkers…
According to a study published in the scientific journal Neurology, the survival rates of patients with Lambert-Eaton myasthenic syndrome (LEMS) were compared to that of the general population as well as…
According to a story from mmm-online.com, a recent infographic highlights the challenges that rare disease patients worldwide still face when it comes to getting the treatment that they so desperately…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
According to a story from curetoday.com, Dr. Tara Seery is an investigator in a phase 3 trial called PROOF, and she has just one goal: finding better treatments for cholangiocarcinoma,…
According to a story from globenewswire.com, the biopharmaceutical company Inventiva is scheduled to present the findings from a recent phase IIa clinical trial at the upcoming 16th Annual WorldSymposium™ which is…
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