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Office
55 Fruit St
# YAW6B
Boston, MA 02114Phone+1 617-726-6093Fax+1 617-726-2019
Education & Training
- Massachusetts General HospitalFellowship, Child Neurology, 2002 - 2005
- Geisinger Health SystemResidency, Pediatrics, 2001 - 2002
- University of Vienna Faculty of MedicineClass of 1997
Certifications & Licensure
- MA State Medical License 2005 - 2026
- PA State Medical License 2001 - 2002
- American Board of Psychiatry and Neurology Neurology with Special Qualification in Child Neurology
Awards, Honors, & Recognition
- Champion of Hope in Rare Diseases Global Genes, 2015
- Teacher of the Year Award in Child Neurology Massachusetts General Hospital, 2013
- Clinical Research Day Departmental Award for Neurology Massachusetts General Hospital, 2011
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Clinical Trials
- A Natural History Study of the Gangliosidoses Start of enrollment: 2010 Dec 01
- L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1 Start of enrollment: 2013 Sep 01
- A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Start of enrollment: 2013 Aug 21
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Publications & Presentations
PubMed
- Anandefficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.Vidyullatha Vasireddy, Casey A Maguire, David W Anderson, Carrie Ng, Yi Gong
Molecular Therapy. Methods & Clinical Development. 2024-12-12 - Urine-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.Amanda Nagy, Florian Eichler, Annette Bley, Janna Bredow, Alexander Fay
Human Gene Therapy. 2024-12-04 - Characterizing visual processing deficits in cerebral adrenoleukodystrophy.Camille S Corre, Melissa Bambery, Christopher R Bennett, Amanda Nagy, Claire E Manley
Brain & Development. 2024-11-01
Press Mentions
- NEJM Reports Detail Blood Cancers After Gene Therapy for Cerebral AdrenoleukodystrophyOctober 16th, 2024
- Gene Therapy for Adrenoleukodystrophy: Studies Find Both Risks and BenefitsOctober 16th, 2024
- Report Gives Update on Boys with Rare Brain Disease 6 Years After Gene TherapyOctober 10th, 2024
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Grant Support
- The Role Of Desoxysphingoid Bases In Hsan1National Institute Of Neurological Disorders And Stroke2011–2012
- 1-Deoxydihydroceramides In Hereditary Sensory &Autonomic Neuropathy Type INational Center For Research Resources2011
- Imaging The Pathophysiology Of AMN In Mice And HumansNational Institute Of Neurological Disorders And Stroke2006–2010
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