FDA grants orphan drug designation to potential treatment of STXBP1 developmental and epileptic encephalopathy
Research that was carried out by Baylor College of Medicine and Texas Children's Hospital doctors has led to the U.S. Food ...
Caliway Announced EMA Orphan Drug Designation Granted to CBL-514 for Dercum's Disease Treatment
Caliway Biopharmaceuticals (Caliway) announced that the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to its novel small molecule drug, CBL-514, for the treatment of Dercum's ...
Immuneering Corp. Reports Q3 Loss, Highlights Progress
Immuneering Corp. Class A ( (IMRX) ) has released its Q3 earnings. Here is a breakdown of the information Immuneering Corp. Class A presented ...
The Pharma Letter4d
FDA’s Orphan Products Grants Program awards significant funding for rare diseases
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases ...
Business Insider4d
Attralus Therapeutic AT-02 Receives Orphan Drug Designation from the U.S. FDA for the Treatment of ATTR Amyloidosis
“We are pleased to have been granted orphan drug designation by the U.S. FDA for AT-02 in ATTR amyloidosis,” said Gregory Bell, M.D., Chief Medical Officer at Attralus. “Current approved therapies for ...
Monthly Prescribing Reference3d
Pan-Amyloid Removal Therapeutic Gets Orphan Drug Status for Systemic Amyloidosis
The Food and Drug Administration (FDA) has granted Orphan Drug designation to AT-02 for the treatment of transthyretin-associated amyloidosis (ATTR). AT-02 is a fusion protein of humanized ...
Pharmabiz1d
Caliway’s drug molecule CBL-514 to treat Dercum’s disease receives EMA orphan drug designation
Caliway’s drug molecule CBL-514 to treat Dercum’s disease receives EMA orphan drug designation: New Taipei City Thursday, November 14, 2024, 16:00 Hrs [IST] Caliway Biopharmac ...
Yahoo Finance4d
Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-108 (HSV-1 oncolytic virus) to Treat Malignant Glioma
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis ...