The biotechnology company ContraFect has entered into an agreement with the Cystic Fibrosis Foundation. This funding agreement aims to investigate and develop direct lytic agents (DLAs) as a treatment for…
Protalix BioTherapeutics has recently completed the treatment portion of their Phase III BRIGHT trial, which is investigating PRX-102 as a treatment for Fabry disease. The data is still being collected…
COVID-19 turned our worlds upside down back in March, and it has been the main focus for many medical professionals since. Because it is a novel coronavirus, we did not,…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
Living with a rare disease can be difficult. Carlos, who has myositis, knows all about this. His mobility has been declining, and he is now in a wheelchair. His doctors…
BioMarin Pharmaceutical Inc. has recently submitted a new drug application (NDA) for their achondroplasia treatment, vosoritide. This once a day injection is for children who live with the disorder, and…
An Australian royal commission is investigating claims that children with disabilities are being excluded from online learning, according to The Guardian. They are also looking into issues with the services…
According to Parkinson's News Today, diet is something that should be monitored in Parkinson's disease patients. Many do not eat enough iron, vitamin B1, zinc, or vitamin D, which was…
A study conducted at the University of Texas has discovered that combination therapy may be a viable treatment for cholangiocarcinoma with BRAF mutations. Researchers treated participants with both dabrafenib and…
I-Mab, a biopharmaceutical firm, has begun dosing participants in Phase Ib of their trial of plonmarlimab, a rheumatoid arthritis (RA) treatment. Developers of the medication believe that it could become…
According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…
Arrowhead Pharmaceuticals has began dosing patients in its AROENaC1001 trial, which evaluates ARO-ENaC as a treatment for cystic fibrosis. 54 patients will be treated with this therapy in an effort…
Finding the best care for every cancer patient can be difficult, as every patient has different needs. Precision care aims to provide the best treatment for every patient, meaning that…
Seelos Therapeutics has recently received approval to proceed with phase IIb/III of their trial of trehalose, an investigational ALS treatment. The FDA alerted Seelos of this approval on August 7…
Biogen and Denali Therapeutics are working together to develop DNL151, a treatment for Parkinson's disease. This medication will move into late-stage trials after positive data from ongoing Phase 1/1b studies.…
The HEALY ALS Platform Trial, being conducted by Biohaven Pharma, has begun enrolling patients. This study is intended to test the efficacy of three treatments on amyotrophic lateral sclerosis (ALS),…
CymaBay Therapeutics has recently released the results from ENHANCE, a study of seladelpar as a treatment for primary biliary cholangitis (PBC). These results were positive, proving that this medication has…
Alnylam Pharmaceuticals has submitted their clinical trial authorization (CTA) application to the Medicines and Healthcare Products Regulatory Agency (MHRA) for their RNAi therapy, ALN-HSD, a treatment for nonalcoholic steatohepatitis (NASH).…
Living with a rare disease can be difficult for a myriad of reasons, one of them being financial. Treatments, appointments, tests, and everything else that comes with a rare disease…
Chronic pain affects about two-thirds of those with Parkinson's, making it the major non-motor symptom of the disease. A study published in Movements Disorders has found that anandamide and glucocerebroside…
INO-3107, a treatment for recurrent respiratory papillomatosis (RRP), has recently received the Orphan Drug Designation from the FDA. This treatment is currently being evaluate in a Phase 1/2 trial by…
According to GlobeNewswire, BridgeBio Pharmaceuticals and the Ivy Brain Tumor Center have recently began their Phase 0/2 trial of infigratinib. This treatment is meant for those with recurrent glioblastoma that…
The pandemic has made it difficult to get the same healthcare that we are used to. It is even harder for those who live with a medical condition or serious…
BioMarin has recently submitted a Marketing Authorization Application to the European Medicines Agency for their achondroplasia treatment, vosoritide. As of now, the review of the application will begin in August.…
The Michael J. Fox Foundation has awarded a grant of $750,000 to researchers at the University of California, Los Angeles for their work to improve the Parkinson's disease registry in…
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