The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this…
According to CheckOrphan, Clinuvel Pharmaceuticals recently announced that their drug Scenesse has received a Prescription Drug User Fee Act (PDUFA) date from the FDA, set for July 8th of this…
According to a story by MarketScreener, pharmaceutical company Acer Therapeutics Inc. has submitted a New Drug Application (NDA) for its vascular Ehlers-Danlos syndrome (vEDS) drug Edsivo. The NDA was granted…
People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
Big news out of the FDA last week! Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval…
Just this week, the Food and Drug Administration approved a drug that targets a wide range of cancers based on DNA, rather than where the tumor is in the body. This is being…
Last week the Biotechnology Innovation Organization released a letter to the FDA, commenting on the latter's draft of a document that proposes a new set of guidelines for the treatment…
Happy Friday! Winter is coming and so is FDA news! Take a moment to check out some recent FDA announcements. FDA Approves Triplet Immunotherapy Combination for Multiple Myeloma Big news…
Happy November! As we recover from Halloween and prepare for Thanksgiving, take a moment to check out some recent FDA announcements. FDA grants breakthrough therapy designation to UGN-101 for urothelial…
Happy Halloween! As the spookiest day of the year approaches, we're highlighting FDA announcements from the last week and a half we think you should hear about. Check them out…
If drugs were measured by the quantity of approvals, orphan drugs would make quite a splash. Measured in more financial terms, however, they make up a much smaller portion of…
Happy Friday everyone! The leaves are beginning to change, and so are ongoing activities at the FDA. This week, we're highlighting four FDA announcements we think you should hear about.…
Happy October, everyone! We hope everyone's enjoying this new ~fall weather~ and the endless supply of pumpkin spice flavored food products. This week, we're highlighting four FDA announcements we think…
Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old. Sutton is now 28 years old. Because of her condition, she must adhere to a very…
Happy Friday, everyone! We're trying something new this week. Instead of wrapping up with an Editor's Choice article, we're highlighting four FDA announcements we think you should know. Check them…
Discussion on Right to Try legislation continues, and the federal government could soon change existing rules. Many people wonder whether or not patients should have the right to try potentially…
New medicines for Parkinson’s may be around the corner. The United States Food and Drug Administration (US FDA) recently approved one drug, and accepted application another. Both new drugs treat…
The FDA has doubled down on warnings regarding balloon angioplasty. Proposed as a treatment for multiple sclerosis, the surgical use of balloons in the jugular vein has been deemed risky…
The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…
Great news for the rare disease community! Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH). XLH…
Lilia Zaharieva was a University of Victoria student in her early late twenties when she found herself losing half her leg function due to cystic fibrosis. Walking from class to…
A major first step for a viable treatment option! Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy…
Earlier this month, the FDA approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. The final results indicated a response rate of 54.5%. This is the first FDA-approved treatment…
Big news for those with hemophilia! On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing…
Sam and Anna Beiler have not one, but two children with a rare degenerative disease that causes blindness. As of now, there is no treatment. Last month, however, the FDA…
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