According to a story from CRWE World, the drug company BioMarin Pharmaceutical, Inc. recently announced that Palynziq, a treatment developed by the company for phenylketonuria, has earned marketing authorization from…
According to a story from fox43.com, an experimental therapy for the genetic form of amyotrophic lateral sclerosis (ALS) has been termed "game changing." A study that will soon be presented…
According to a story from EurekAlert!, the biopharmaceutical company Celgene Corporation and the gene therapy company bluebird bio, inc. recently announced that the interim results for a Phase 1 clinical…
According to a story from globenewswire.com, the pharmaceutical company Acer Therapeutics recently announced the release of data from a long-term observational study of patients with vascular Ehlers-Danlos syndrome (vEDS). All…
.jpg)
The News Hub at Washington State University recently carried an article describing initial success by researchers towards developing a drug for a rare form of muscular dystrophy. Several thousand people…
According to a story from finanzen.at, a recent early stage clinical study showed positive results from an innovative stem cell therapy treatment. In the study, this therapy was able to…
According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…
According to a story from Healio, a recent study has found that 58 percent of patients with the inflammatory disease ankylosing spondylitis will experience relapse within one year after stopping…
According to a story from finanzen.ch, the pharmaceutical company Eisai, Inc. announced that that company presented a total of four abstracts which were related to a Phase 3 clinical trial…
According to a story from The Del Mar Times, the Susan Poorman Blackie Ovarian Cancer Foundation (SPB Foundation) and the Clearity Foundation have recently partnered in order to facilitate a…
According to a story from express.co.uk, Elle Morris was only 11 years old when she died from a severe lung infection. Elle was born with cystic fibrosis, a serious genetic…
According to a story from the Edinburgh Evening News, Stephen Ritchie of Edinburgh, Scotland, has been an active participant in a progressive multiple sclerosis clinical trial since November of last…
Noonan Syndrome Noonan syndrome (NS) is a rare disorder that is typically diagnosed at birth. It affects the child's development resulting in stunted growth, abnormal facial features, and heart defects,…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc recently announced encouraging results from an interim safety review of the company's Phase 2a clinical trial. This…
According to a story from Healthworld, the government of Kerala, an Indian state located on the southwestern coast, has announced plans to take action in order to treat patients with…
According to a story from globenewswire.com, the biotechnology company Biogen recently released data from an open label study that was testing the company's spinal muscular atrophy drug nusinersen (marketed as…
.jpg)
According to an article in Biospace, the FDA is currently reviewing data from the Phase III STRIVE clinical trial for possible approval of Zolgensma as gene therapy for spinal…
According to a press release from Massachusetts-based biotech company Fulcrum Therapeutics, the company has successfully secured global commercialization rights of GlaxoSmithKline-developed experimental facioscapulohumeral muscular dystrophy (FSHD) drug losmapimod. Losmapimod, an…
According to a publication from the BBC, a novel new technology could help some with Parkinson's disease regain limited walking ability. However, unlike medical technology we often showcase on Patient…
According to a publication from Healio, recent breakthroughs in medical science have renewed hopes of finding a cure for the rare Cushing disease. Dr. Shlomo Melmed, dean and executive Vice…
.jpg)
Just one year ago David Chen, a forty-year-old policeman who lives in Toronto, was walking home after playing volleyball. According to a recent article in CBC Canada News, a…
There is a new show on television that showcases the experiences of patients and doctors at the Queen Elizabeth Hospital in Birmingham. On its first premiere, it showed the…
According to a story from BioSpace, the biotechnology company Viela Bio recently announced that the US Food and Drug Administration (FDA) has awarded an experimental therapy in development by the…
According to a story from EurekAlert!, effective management of Parkinson's disease can be a serious challenge. Part of the reason that the management of the illness is so difficult is…
According to a story from globenewswire.com, the gene therapy company Fibrocell Science, Inc. and the biopharmaceutical company Castle Creek Pharmaceuticals have recently announced a collaboration between the two companies. The…
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.
