Continued progress in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) toward first subject treated, on track to take place in the first quarter of 2025 - Multiple subjects currently ...
Continued progress in pivotal, adaptive Phase 3 clinical trial (the “MIRACLE” trial) toward first subject treated, on track to take place in ...
She was a little girl. Her world was turned upside down. But she maintained her determination and hopefulness,” said friend and author Kristen Arute ...
Soleno Therapeutics is nearing FDA approval for its lead drug, DCCR, targeting Prader-Willi Syndrome, a rare genetic ...
No country in Europe is as much a product of enlightened postwar American diplomacy. Now adrift, it has begun to reckon with ...
Rhythm Pharmaceuticals received an orphan-drug designation from Japan's Ministry of Health, Labour and Welfare for its treatment for a certain type of obesity.
HTA as described by the HTA Regulation is the multidisciplinary scientific evidence-based process that allows to determine the relative effectiveness of new or existing health technologies. HTA is a ...
“Orphan drug designation points to the need for therapeutic ... “With this designation now in place in Japan, as well as in Europe, we believe we are well positioned to execute on our global strategy ...
INmune Bio's high-upside potential with CORDStrom for RDEB and XPro for Alzheimer's, offering up to 144.8x returns. Click ...
Namodenoson, has been also granted Orphan Drug Designation by the FDA for pancreatic cancer. The designation as an orphan drug will provide, among others, potential for market exclusivity for seven ...
Attendees will gain insights into key market access, pricing and reimbursement challenges in Europe. The featured ... access opportunities If involved in orphan drug development, the attendees ...
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