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Dr. Jonanlis Ramirez Alcantara presents at the 2022 Pediatric Endocrine...
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,...
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Natalie Grant presents at 2022 AAN Meeting
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...
View ArticlePoxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the...
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...
View ArticleSwanBio Therapeutics Announces $56 Million Series B Financing to Advance...
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...
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FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral...
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...
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Minoryx‘s Marketing Authorization Application for its lead candidate...
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,...
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bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy 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...
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Communication and testing experiences of adolescent girls and adult women in...
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...
View ArticleMinoryx announces enrollment of first patients with cerebral...
Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYX 2023-11-16 As part of CALYX, Minoryx is conducting an extensive...
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