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Featured
Innovations in ALS Clinical Trials
Merit Cudkowicz, MD, MSc, chief of Neurology at Massachusetts General Hospital, discusses innovations in ALS clinical trials since she started in the field in 1994.
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Featured
Advancements in the Treatment of Neurodegenerative Disease
In this video, Ghazaleh Sadri-Vakili, PhD, director of the NeuroEpigenetics Laboratory and the MassGeneral Institute of Neurodegenerative Disease, discusses her genetic research, specifically concerning neurodegenerative diseases like ALS and X-linked dystonia parkinsonism.
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Featured
Identifying New Potential Therapeutic Targets for ALS FTD
Transcripts of repeat expansions in the C90RF72 gene undergo a repeat-associated non-AUG (RAN) translation pathway that generates multiple aberrant proteins. Researchers at Mass General Hospital identified new potential therapeutic targets for ALS FTD.
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Antisense Drugs Mimic TDP-43 Function, Might Treat ALS and Some Dementias
Clotilde Lagier-Tourenne, MD, PhD, and colleagues have demonstrated in vitro and in vivo efficacy of antisense oligonucleotides in preventing missplicing of stathmin-2 in a mouse model of amyotrophic lateral sclerosis attributable to deficiency of the protein TDP-43.
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Design and Statistical Innovations in the Healey ALS Platform Trial
Sabrina Paganoni, MD, PhD, Eric A. Macklin, PhD, and colleagues explain the statistical approaches used in the Healey ALS Platform Trial to integrate functional and survival outcomes and account for potential differences in the outcomes of participants within the shared control group.
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Genomic Therapeutics in Action: Antisense Oligonucleotides Interventions Reverses Neurodegeneration
Physician-investigators at Massachusetts General Hospital recently published findings that provide a critical step forward in understanding the mechanisms underlying a host of neurodegenerative diseases.
ALS Contributors
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Clotilde Lagier-Tourenne, MD, PhD
Associate Professor of Neurology, Massachusetts General Hospital and Harvard Medical School
Recent Article
Antisense Drugs Mimic TDP-43 Function, Might Treat ALS and Some Dementias
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Ghazaleh Sadri-Vakili, MS, PhD
Assistant in Neuroscience, Massachusetts General Hospital, Assistant Professor of Neurology, Harvard Medical School
Recent Article
Novel Target Identified for Treatment of ALS
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James D. Berry, MD, MPH
Co-director, Multidisciplinary Amyotrophic Lateral Sclerosis (ALS) Clinic
Recent Article
Healey Platform ALS Trial Expected to Expedite U.S. Approval of New Treatments
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Merit Ester Cudkowicz, MD, MSc
Chief, Neurology Service, Director of the Sean M. Healey & AMG Center for ALS at Mass General, Director of Neurological Clinical Research Institute and the Jilieane Dorn Professor of Neurology, Harvard Medical School
Recent Article
ALS Therapy Research and Development: Insights from Merit Cudkowicz, MD, MSc
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Nazem Atassi, MD
Associate Director, Neurological Clinical Research Institute (NCRI), Mass General Hospital, Associate Professor of Neurology, Harvard Medical School
Recent Article
High-Dose Tamoxifen "Wins" Selection Trial in ALS
Neuroscience Care at Mass General
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