According to a story from BioSpace, the genomic medicine company Sangamo Therapeutics, Inc. and Pfizer, Inc. recently announced the release of interim data from a Phase I/II clinical trial. This…
.jpg)
A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
.jpg)
A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
.jpg)
Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
According to the publication from RedlionTrader, a second man infected with HIV seems to have been cleared of the infection, those scientists remain highly cautious of touting the case as…
According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company's clinical trial. This is a…
The National Institutes of Health (NIH) is the primary medical research institution in the United States. The NIH is one of several public health institutions, like the Food and Drug…
According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive…
According to a press release written by French biotech company Deinove and published by PM360, the pharmaceutical manufacturer stands ready to begin phase 2 testing into their experimental Clostridium difficile…
According to a story from Myeloma Research News, the UK National Institute of Health and Care Excellence (NICE) has recently decided to approve a three part combination treatment for coverage…
According to a story from PR Newswire, the biopharmaceutical company Therachon AG recently issued a presentation of results from the company's Phase I clinical trial. This clinical trial tested the…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…
According to a story from globenewswire.com, the biopharmaceutical company Strongbridge Biopharma plc, recently presnted a new analysis of data from the company's Phase 3 trial. This trial was testing the…
According to a story from BioPortfolio, the biopharmaceutical company AlzProtect recently announced that PAREXEL Biotech, which is a subsidiary of PAREXEL International Corporation, will be expected to conduct a Phase…
According to a story from finanzen.ch, the drug companies Pfizer and Merck recently released a joint statement in which they announced that the companies' Phase III clinical trial would be…
.jpg)
A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…
.jpg)
A recent article in Parkinson’s News Today reports that a reduction in a patient’s required medication and management of motor system disorders may be achievable with Deep-Brain Stimulation (DBS) for…
.jpg)
Following a record number of approvals in 2018, the FDA has recently approved two new drugs with many more anticipated approvals later in the year. Trazimera to Treat Human Epidermal…
According to a publication from Multiple Sclerosis News Today, German biotechnology company Immunic Therapeutics has successfully enrolled the first participant for its phase 2 clinical trial for experimental relapsing-remitting multiple…
According to a story from Markets Insider, the biopharmaceutical company Orchard Therapeutics recently announced the presentation of proof-of-concept data in regards to their investigational drug candidate OTL-102. This experimental therapy…
According to a story from Nursing Times, Claire Naisbitt is one of three multiple sclerosis specialist nurses that treat patients that reside in the Teesside community. She first took the…
According to a recent article in the Globe, the Danish biotech company Saniona AB has initiated a Phase 2a clinical trial of Tesomet for the treatment of hypothalamic obesity, a rare…
According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…
According to a story from globenewswire.com, the drug developer The Cell Factory and the University of Padova in Italy have been conducting research in order to demonstrate the effectiveness of…
According to a story from Market Screener, the biopharmaceutical company Marinus Pharmaceuticals, Inc. recently announced its plans to initiate a Phase 3 study that is intended to test ganaxolone as…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
