Moleculin Biotech, Inc.: Moleculin Reports Full Year 2024 Financial Results and Provides Corporate Update
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 ...
Moleculin Reports Full Year 2024 Financial Results and Provides Corporate Update
Continued progress in pivotal, adaptive Phase 3 clinical trial (the “MIRACLE” trial) toward first subject treated, on track to take place in ...
Soleno Therapeutics: FDA Approval May Prove To Be A Pyrrhic Victory
Soleno Therapeutics is nearing FDA approval for its lead drug, DCCR, targeting Prader-Willi Syndrome, a rare genetic ...
In Germany, ‘Orphaned’ by U.S., Shock Gives Way to Action
No country in Europe is as much a product of enlightened postwar American diplomacy. Now adrift, it has begun to reckon with ...
The Lancet4d
Advancing policies for rare diseases in Europe
Fostering dialogue between neurologists, researchers, patient representatives, and policymakers is an important step towards ...
Capricor Therapeutics Stock (CAPR) Surges on Q4 Results and Promising New Therapy
Capricor Therapeutics (CAPR) released expectation-beating results for the fourth quarter, sending the shares up over 5% in aftermarket trading.
Rhythm Pharmaceuticals Receives Orphan-Drug Designation in Japan for Obesity Treatment
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.
JD Supra4d
Early benefit assessment of medicinal products in Germany – first adaptions to AMNOG implementing the EU HTA Regulation
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 ...
Yahoo Finance5d
Rhythm Pharmaceuticals Announces Orphan Drug Designation Granted to Setmelanotide for Treatment of Hypothalamic Obesity in Japan
“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: CORDStrom Is A Game-Changer, As Investors Await Alzheimer's Results
INmune Bio's high-upside potential with CORDStrom for RDEB and XPro for Alzheimer's, offering up to 144.8x returns. Click ...
TMCnet6d
Can-Fite: FDA Approved Compassionate Use Treatment with Namodenoson in a Pancreatic Cancer Patient
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 ...