BioMarin: Phase 1/2 Study On Hemophilia A Shows Hemostatic Control With Valoctocogene Roxaparvovec
WASHINGTON (dpa-AFX) - BioMarin Pharmaceutical Inc. (BMRN), in an update on phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, said Wednesday that the safety profile of valoctocogene roxaparvovec in the study remains consistent with previously reported data with no delayed-onset treatment related adverse events.
The company noted that the latest data update in the ongoing study represents the longest duration of clinical experience for any gene therapy in hemophilia A and demonstrates hemostatic control with valoctocogene roxaparvovec out to five years in the majority of patients in this study.
Y-mAbs Enters into Exclusive Distribution Agreement with Adium Pharma S A for DANYELZA® (naxitamab-gqgk) and Omburtamab in Latin America morningstar.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from morningstar.com Daily Mail and Mail on Sunday newspapers.
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EQS Group-News: RELIEF THERAPEUTICS Holdings AG / Key word(s): Miscellaneous
18.05.2021 / 07:00
Geneva, Switzerland, May 18, 2021 - RELIEF THERAPEUTICS Holding AG (SIX: RLF, OTCQB: RLFTF)(
Relief ), a biopharmaceutical company with its lead compound RLF-100
TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, today provided an update on the development of ACER-001, a proprietary powder formulation of sodium phenylbutyrate (NaPB) designed to be both taste-masked and immediate release, in the lead indication, urea cycle disorders (UCDs). UCDs are a group of rare genetic metabolic disorders which can lead to an excess accumulation of ammonia in the bloodstream, causing different symptoms such as somnolence, coma, and, in the worst case, may lead to multi-organ failure. Better, more affordable treatment options for UCDs are urgently needed.
Relief ), a biopharmaceutical company with its lead compound RLF-100
TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, today provided an update on the development of ACER-001, a proprietary powder formulation of sodium phenylbutyrate (NaPB) designed to be both taste-masked and immediate release, in the lead indication, urea cycle disorders (UCDs). UCDs are a group of rare genetic metabolic disorders which can lead to an excess accumulation of ammonia in the bloodstream, causing different symptoms such as somnolence, coma, and, in the worst case, may lead to multi-organ failure. Better, more affordable treatment options for UCDs are urgently needed.
Photocure announces Hexvix regulatory filing accepted for review in Chile with Fast Track designation prnewswire.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from prnewswire.com Daily Mail and Mail on Sunday newspapers.