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Genetic Disorders

Latest News

February 5th 2020
Ataluren Extends Ability to Walk in Patients With Duchenne Muscular Dystrophy

Interim analysis compared ataluren in a real-world setting from the STRIDE registry with a matched cohort; the final data from the STRIDE registry are expected in 2025.

Genetics DNA genetic testing
July 3rd 2019

Genetic Testing for Huntington Disease

Angelman syndrome researcher
June 25th 2019

Phase 2 Trial for Angelman Syndrome Underway

CME Content

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Primary Care Education in Nashville
In-Person + Virtual Event

Primary Care Education in Nashville

October 20-21, 2026

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Shining a Light on an Ultra-Rare Disease – A Closer Look at Thymidine Kinase 2 Deficiency (TK2d)
Multimedia

Shining a Light on an Ultra-Rare Disease – A Closer Look at Thymidine Kinase 2 Deficiency (TK2d)

Zarazuela Zolkipli-Cunningham, MBChB, MRCP; Mary Kay Koenig, MD; Perry Shieh, MD, PhD, FAAN, FANA

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Expert Illustrations & Commentaries™: Visualizing the Role of Dystrophin Dysregulation as a Therapeutic Target in Duchenne Muscular Dystrophy and MD STARnet
Video

Expert Illustrations & Commentaries™: Visualizing the Role of Dystrophin Dysregulation as a Therapeutic Target in Duchenne Muscular Dystrophy and MD STARnet

Anne M. Connolly, MD, FAAN; Amy Moore, PhD

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Show Me the Data™: Turning Molecular Drivers Into Precision Care for CNS Tumors
In-Person + Virtual Event

Show Me the Data™: Turning Molecular Drivers Into Precision Care for CNS Tumors

November 12, 2026

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Putting the Patient First in Acute Pain Management: The PA’s Guide to Incorporating Cutting-Edge Science Into Their Treatment Strategies
Video

Putting the Patient First in Acute Pain Management: The PA’s Guide to Incorporating Cutting-Edge Science Into Their Treatment Strategies

Jessica Oswald, MD, MPH; Jeremy A. Adler, DMSc, PA-C; Amanda Nasser, MS, NP

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Expert Illustrations & Commentaries™: Visualizing the Role of B Cells as Therapeutic Targets for Generalized Myasthenia Gravis
Video

Expert Illustrations & Commentaries™: Visualizing the Role of B Cells as Therapeutic Targets for Generalized Myasthenia Gravis

James F. Howard Jr, MD, FAAN; Katherine Ruzhansky, MD, MS

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Unlocking the Future of Glioma Care: Integrating Recent Advances to Personalize Treatment
Multimedia

Unlocking the Future of Glioma Care: Integrating Recent Advances to Personalize Treatment

Vinay K. Puduvalli, MD; Manmeet Ahluwalia, MD, MBA, FASCO; Girish Dhall, MD

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From Clue to Care: Rapid Recognition and Coordinated Management of Paraneoplastic LEMS in SCLC
Video

From Clue to Care: Rapid Recognition and Coordinated Management of Paraneoplastic LEMS in SCLC

Millie Das, MD; Wade Iams, MD, MSCI; Ruham Nasany, MD

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Collaborating Across the Continuum™: Integrating Novel Therapies Into Multidisciplinary Treatment Plans for Generalized Myasthenia Gravis
Video

Collaborating Across the Continuum™: Integrating Novel Therapies Into Multidisciplinary Treatment Plans for Generalized Myasthenia Gravis

Abigail Schwaede, MD; Alexis Lizarraga, MD, MS

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Community Collab: The Evolving Role of FcRN Inhibitors in the Management of Generalized Myasthenia Gravis (gMG)
Multimedia

Community Collab: The Evolving Role of FcRN Inhibitors in the Management of Generalized Myasthenia Gravis (gMG)

James F. Howard Jr, MD, FAAN; Tuan Vu, MD

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From Suspicion to Stabilization: Early Recognition and Treatment of Paraneoplastic LEMS
Video

From Suspicion to Stabilization: Early Recognition and Treatment of Paraneoplastic LEMS

Charu Aggarwal, MD, MPH, FASCO; Erminia Massarelli, MD, PhD, MS; Ruham Nasany, MD

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How Do I Identify MOGAD? Improving Outcomes Through Early Identification of Patients
Video

How Do I Identify MOGAD? Improving Outcomes Through Early Identification of Patients

Michael Levy, MD, PhD; Jeffrey L. Bennett, MD, PhD; Eoin P. Flanagan, MB, BCh

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