29th September 2022

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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 both a patient and their biological relatives. While testing in some circumstances is considered routine and consistent … Continued

17th September 2022

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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 neurologic dysfunction in boys with this devastating and fatal neurodegenerative disease Management team to host conference call … Continued

14th September 2022

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Press Releases

Minoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD)

Mataró, Barcelona, Spain, September 14, 2022 – Minoryx Therapeutics, a late-stage biotech company focused on the development of treatments for orphan central nervous system (CNS) disorders, today announces that it … Continued

9th June 2022

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FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy

Research study: Lorem ipsum dolor sit amet, per appetere percipit disputationi et, homero nostrud vituperatoribus in duo, id pro tota nobis.

18th May 2022

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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). New funding will support ongoing clinical...

17th May 2022

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Press Releases

Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy

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 … Continued

News & Events

Communication and testing experiences of adolescent girls and adult women in families with X-linked adrenoleukodystrophy

bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD)

Minoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD)

FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy

SwanBio Therapeutics Announces $56 Million Series B Financing to Advance Novel Gene Therapies for Neurological Conditions

Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy

Raise Awareness for ALD

News & Events

Communication and testing experiences of adolescent girls and adult women in families with X-linked adrenoleukodystrophy

bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD)

Minoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD)

FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy

SwanBio Therapeutics Announces $56 Million Series B Financing to Advance Novel Gene Therapies for Neurological Conditions

Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy

Raise Awareness for ALD

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