The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…
According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing…
by Danielle Bradshaw from In The Cloud Copy Bone grafts, although very helpful, can result in hard to treat infections and typically result in the patient needing to be put…
United Therapeutics Corporation has recently presented data from the Phase 3 INCREASE trial, which evaluated Tyvaso as a treatment for pulmonary hypertension associated with interstitial lung disease. Results were presented…
According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…
According to a story from UT Health San Antonio, a team of researchers have discovered in a new study the mechanism behind the activation of magnesium ions in cells. The…
According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…
After announcing positive results from a Phase 2b ADVISE trial, Arena Pharmaceuticals, Inc. ("Arena") hopes to soon initiate a Phase 3 clinical trial to evaluate etrasimod as a treatment…
The European Medicines Agency has accepted Biogen's Marketing Authorization Application (MAA) for review. The application is for aducanumab, a treatment for Alzheimer's disease. As this condition faces an unmet medical…
In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…
The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to…
According to GlobeNewswire, the results from a Phase 1 study of lirentelimab have been released. Allakos developed this medication as a treatment for eosinophilic and mast-cell related diseases. In this…
According to a story from Medscape, a recent large scale trial focused on cancer treatment has produced some interesting results. The trial was aimed at testing next generation sequencing of…
Charcot-Marie-Tooth (CMT) disease is a neurological disorder with a number of subtypes. Because of this, no treatment is universally successful within all affected individuals. In order to get patients the…
Blood cancer treatment is undergoing a transformation, according to a recent study in Nature Medicine. In this regard, the Leukemia & Lymphoma Society (LLS) recently published its own account featuring…
Evrysdi, a treatment for spinal muscular atrophy (SMA), has started to make progress around the world in terms of approval. Brazil's regulatory agency, National Health Surveillance Agency, has approved the…
According to a story from GlobeNewswire, the medication dupilumab (marketed as Dupixent®) has demonstrated potential efficacy in a phase 3 clinical trial as a treatment for eosinophilic esophagitis, a rare…
A recent article published in Multiple Sclerosis News Today reported that newly identified immune cells prevented the death of injured nerve cells. A study finds that this particular immune cell…
Medical professionals are constantly looking for treatments that will halt or even reverse the progression of Alzheimer's disease, and a new vaccine called E22W42 may be a large step in…
A recent study, published in the journal Medicine, has found that Mycopheenolate mofetil (MMF) could be a more advantageous therapy for lupus nephritis than cyclophosphamide (CYC) when it comes to improving serum complement…
Many rare diseases are in dire need of need of viable treatments. To fix this problem, researchers have began creating and indicating existing treatments for rare diseases. An example would…
According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…
An article recently published in Genetic Engineering & Biotechnology News, heralds DrugCell, an experimental artificial intelligence (AI) system. DrugCell was created at the Moores Cancer Center and the School of…
According to a story from Biotech 365, the biotechology company Kezar Life Sciences, Inc. recently announced that its experimental therapy KZR-616 has earned Orphan Drug designations from the US Food…
A new treatment for Krabbe disease, PBKR03, has recently been granted the Orphan Drug and Rare Pediatric Disease designations by the FDA, according to an article in GlobeNewswire. These designations…
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