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Office
300 Longwood Ave
Boston, MA 02115Phone+1 617-355-6394Fax+1 617-252-7694
Summary
- I am a board-certified clinical geneticist who has been developing drugs to treat rare genetic diseases for over 20 years. Most of my industry experience was in clinical development at Genzyme, where I contributed to the approvals of several drugs for lysosomal storage disorders, including Gaucher and Pompe diseases, and MPS I and MPS II. From 2016-2018, I was CMO at Editas Medicine, where I led the first IND for a CRISPR medicine delivered in vivo to treat a form of genetic blindness (LCA10). For the past two years, I have been consulting for several biotech companies developing innovative drugs for rare genetic diseases with therapeutic approaches that span all the way from small molecules to enzymes, proteins, antibodies, gene therapy, and genmoe editing. I serve on the Board of Directors for the National Tay-Says and Allied Diseases organization, where I have been helping families understand the clinical trial process and how they can contribute to researching treatments and cures. Throughout my career, I have always maintained a continuity clinic in clinical Genetics at Boston Children's Hospital - some of the infants with formerly fatal diseases are now all grown up and entering their 30's.
Education & Training
- Boston Children’s Hospital/Harvard Medical SchoolFellowship, Medical Genetics and Genomics, 1992 - 1994
- Boston Children’s Hospital/Boston Medical CenterResidency, Pediatrics, 1989 - 1992
- University of California San Diego School of MedicineClass of 1989
Certifications & Licensure
- MA State Medical License 1992 - 2024
- American Board of Medical Genetics and Genomics Clinical Biochemical Genetics
- American Board of Pediatrics Pediatrics
- American Board of Medical Genetics and Genomics Clinical Genetics and Genomics
- American Board of Medical Genetics and Genomics Clinical Molecular Genetics and Genomics
Awards, Honors, & Recognition
- Howard Hughes Physician Scientist Fellowship Howard Hughes, 2014-2017
Publications & Presentations
PubMed
- 122 citationsA prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B.Margaret M. McGovern, Melissa P. Wasserstein, Roberto Giugliani, Bruno Bembi, Marie T. Vanier
Pediatrics. 2008-08-01 - 22 citationsBurden of Illness in Acid Sphingomyelinase Deficiency: A Retrospective Chart Review of 100 PatientsGerald F. Cox, Lorne A. Clarke, Roberto Giugliani, Margaret M. McGovern
JIMD Reports. 2018-07-12 - 603 citationsIncidence, causes, and outcomes of dilated cardiomyopathy in children.Jeffrey A. Towbin, April M. Lowe, Steven D. Colan, Lynn A. Sleeper, E. John Orav
JAMA. 2006-10-18
Press Mentions
- Cyclo Therapeutics Appoints Lise Lund Kjems, MD, PhD as Chief Medical OfficerSeptember 27th, 2021
- Lise Lund Kjems, MD, PhD as Chief Medical OfficerSeptember 27th, 2021
- Cyclo Therapeutics to Present at the JMP Securities Life Sciences ConferenceJune 9th, 2021
- Join now to see all
Grant Support
- Leptin Levels In Children With Syndromic And Nonsyndromic ObesityNational Center For Research Resources1998–2000
- Homozygosity Mapping Of A Bardet Biedl Syndrome Gene In Families Of PR AncestryNational Center For Research Resources1997–1999
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