RespireRx Pharmaceuticals announced last month it was cleared to resume its ampakine CX717 clinical trials to evaluate the drug’s efficacy in several central nervous system and respiratory disorders, including Pompe…
Last month, the FDA has granted Achillion Pharmaceuticals orphan drug designation to ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G). According to the FDA, the Orphan Drug Designation program provides…
Nothing is worse for a family than hearing the word “cancer,” especially when it involves a child. The Tyson family was ready to start their perfect life eleven years ago.…
New research is being conducted to develop new technologies to detect ovarian cancer. Keep reading or follow the original story here to learn more about the struggles and developments involved…
According to a story from the National Hemophilia Foundation, the Swedish pharmaceutical company Orphan Biovitrum announced that its first patient was registered for a new phase 4 clinical trial. The…
According to an article from businesswire.com, the pharmaceutical company Odonate Therapeutics announced the beginning of CONTESSA, a phase three study of the treatment tesetaxel and its function in patients with…
Patients suffering from sickle cell disease, SCD, might just be getting news they've been waiting to hear for a long time. Just this week, the U.S. Food and Drug Administration…
While it's general knowledge that working out is a huge plus for a healthy life, many questioned whether it was positive or dangerous to individuals with rare diseases. Yet, BMC…
According to a story from financialbuzz.com, the pharmaceutical company AveXis, Inc., is planning to begin preparations to submit a Biologics License Application (BLA) to the Food and Drug Administration for…
Watch Prof. Charles Craddock discuss important and heartening results he and his team presented at the American Society of Hematology 2017 Annual Meeting and Exposition in Atlanta, GA - regarding whether there…
According to a story from prnewswire.com, the experimental treatment SPR001 was just granted orphan drug status by the Food and Drug Administration (FDA). The new treatment is currently being developed…
Nikki Oosthuizen gave her 16-year-old daughter, Kim, a kidney as she suffers from a rare kidney disease, yet only four days later the disease returned and destroyed her kidney function…
2017 was a challenging year for 10-year-old Zach Pickard, but at first glance of his smile and rolling laughter, you would never expect it. Zach is among 144 children in…
Cycle Pharmaceuticals shook hands with US-based firm, Aprecia Pharmaceuticals as they agreed to develop and commercialize treatments for rare diseases using the advanced technology of 3D printing, recently reported Cambridge…
Gene therapy has successfully made its presence known in the medical field this last year, reported ABC News. Dr. Francis Collins of the National Institutes of Health isn't afraid to…
A recent study has revealed a possible mechanism for the repair of nerves damaged by multiple sclerosis. Research at the University of California has found that gene expression in certain…
Organovo Holdings recently stated that its 3D bioprinted therapeutic liver tissue was granted orphan drug status by the Food and Drug Administration (FDA). Taylor Crouch, the company CEO, was excited…
'Tis the season for gene therapy approvals. Reported by Bloomberg, the U.K. just approved its most expensive medication to date. The treatment is for severe combine immune deficiency, or more…
The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed…
The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…
Have you been told that you had to stop doing your passion in life because of your rare disease? This is the story of opera lover, Charity Tillemann-Dick. Her life…
DelMar Pharmaceuticals announced that its flagship cancer treatment drug, called VAL-083, was given "Fast Track" status by the Food and Drug Administration. VAL-083 is a possible treatment for a type…
According to an article at publicnow.com, the FDA has granted Breakthrough Therapy Designation to avelumab combined with INLYTA (axitinib) for people with advanced renal cell carcinoma. This designation should accelerate…
Good news for Prometic Life Science new drug application! The pharmaceutical company has been searching for a cure for idiopathic pulmonary fibrosis (IPF), a rare lung disease which you can…
The active and adventurous Devaney Mehl was sidelined by excruciating pain when only 28-years-old, originally reported in Regina Leader-Post. After many incorrect diagnoses, she later learned she had developed the rare…
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