Research Study: Communication and testing experiences of adolescent girls and adult women in families with X-linked adrenoleukodystrophy Purpose of Research: Genetic counselors are often involved in facilitating genetic testing for…
bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD) SKYSONA is the first FDA approved therapy shown to slow the progression of…
Minoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD) 2022-09-14 Mataró, Barcelona, Spain, September 14, 2022 – Minoryx Therapeutics, a late-stage biotech company…
FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a…
SwanBio Therapeutics Announces $56 Million Series B Financing to Advance Novel Gene Therapies for Neurological Conditions New funding will support ongoing clinical development of lead candidate, SBT101 for adrenomyeloneuropathy (AMN)…
Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy LYON, France–(BUSINESS WIRE)– POXEL SA (Euronext: POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative…
Natalie Grant presents at 2022 AAN Meeting Natalie Grant, Clinical Research Coordinator, at Massachusetts General Hospital presented, “Neurological Disease Burden in Females with X-Linked Adrenoleukodystrophy” at the 2022 American Academy…
Dr. Jonanlis Ramirez Alcantara presents at the 2022 Pediatric Endocrine Society Meeting Dr. Jonanlis Ramirez Alcantara, Clinical and Research Fellow at Massachusetts General Hospital, presented, “ADRENAL INSUFFICIENCY IN PEDIATRIC PATIENTS…
Cellular, Tissue and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments Original post by the Food and Drug Administration on Apr 13, 2022 The Food…
