Rare and Orphan Diseases - Global Neurology Academy

Episodes 1-16 of 60

Improving Survival and Function in Pediatric TK2d with Nucleoside Therapy
Improving Survival and Function in Pediatric TK2d with Nucleoside Therapy
Clinician's Roundtable
Improving Survival and Function in Pediatric TK2d with Nucleoside Therapy
- Charles Turck, PharmD, BCPS, BCCCP
- Caterina Garone, MD, PhD
Uncovering the Disease Burden of Untreated Pediatric TK2 Deficiency
Uncovering the Disease Burden of Untreated Pediatric TK2 Deficiency
Clinician's Roundtable
Uncovering the Disease Burden of Untreated Pediatric TK2 Deficiency
- Charles Turck, PharmD, BCPS, BCCCP
- Cristina Domínguez González, MD, PhD
Understanding the Caregiver Burden in TK2 Deficiency: New Insights from a Study
Understanding the Caregiver Burden in TK2 Deficiency: New Insights from a Study
Clinician's Roundtable
Understanding the Caregiver Burden in TK2 Deficiency: New Insights from a Study
- Charles Turck, PharmD, BCPS, BCCCP
- Philip Yeske, PhD
Optimizing Generalized Myasthenia Gravis Care Through Patient-Centered Choices
Optimizing Generalized Myasthenia Gravis Care Through Patient-Centered Choices
NeuroFrontiers
Optimizing Generalized Myasthenia Gravis Care Through Patient-Centered Choices
- Sheetal Shroff, MD
Timing Meningococcal Vaccines in gMG: Challenges and Considerations
Timing Meningococcal Vaccines in gMG: Challenges and Considerations
NeuroFrontiers
Timing Meningococcal Vaccines in gMG: Challenges and Considerations
- Michael Weiss, MD
Individualizing gMG Treatment: Defining Success With Each Patient
Individualizing gMG Treatment: Defining Success With Each Patient
NeuroFrontiers
Individualizing gMG Treatment: Defining Success With Each Patient
- Omar Sinno, MD
Optimizing the Role for Corticosteroids Within the Evolving DMD Treatment Landscape
Optimizing the Role for Corticosteroids Within the Evolving DMD Treatment Landscape
CME/CE
Optimizing the Role for Corticosteroids Within the Evolving DMD Treatment Landscape
1.00 credit
- Stephen Chrzanowski, MD, PhD
- Aravindhan Veerapandiyan, MD
1.00 credit
Advancements in gMG Management: Zilucoplan as an Alternative to C5 Inhibitors
Advancements in gMG Management: Zilucoplan as an Alternative to C5 Inhibitors
NeuroFrontiers
Advancements in gMG Management: Zilucoplan as an Alternative to C5 Inhibitors
Examining Inebilizumab in Generalized Myasthenia Gravis: Insights from MINT
Examining Inebilizumab in Generalized Myasthenia Gravis: Insights from MINT
NeuroFrontiers
Examining Inebilizumab in Generalized Myasthenia Gravis: Insights from MINT
- Ryan Quigley
ASD in Children with SMA Type I: Diagnostic Challenges in a Multifactorial Context
ASD in Children with SMA Type I: Diagnostic Challenges in a Multifactorial Context
NeuroFrontiers
ASD in Children with SMA Type I: Diagnostic Challenges in a Multifactorial Context
Safety and Efficacy of Onasemnogene Abeparvovec in Pediatric SMA Across Expanded Weight Cohorts
Safety and Efficacy of Onasemnogene Abeparvovec in Pediatric SMA Across Expanded Weight Cohorts
NeuroFrontiers
Safety and Efficacy of Onasemnogene Abeparvovec in Pediatric SMA Across Expanded Weight Cohorts
New-Onset Ketosis-Prone Diabetes in an Adult with Spinal Muscular Atrophy Type III
New-Onset Ketosis-Prone Diabetes in an Adult with Spinal Muscular Atrophy Type III
NeuroFrontiers
New-Onset Ketosis-Prone Diabetes in an Adult with Spinal Muscular Atrophy Type III
Reevaluating Spinal Muscular Atrophy Therapy: Signs, Tools, and Strategies
Reevaluating Spinal Muscular Atrophy Therapy: Signs, Tools, and Strategies
NeuroFrontiers
Reevaluating Spinal Muscular Atrophy Therapy: Signs, Tools, and Strategies
- Brian P. McDonough, MD, FAAFP
- Nancy Kuntz, MD
Exploring Circulating CD34+ Cells as a Biomarker of Fracture Risk in DMD
Exploring Circulating CD34+ Cells as a Biomarker of Fracture Risk in DMD
NeuroFrontiers
Exploring Circulating CD34+ Cells as a Biomarker of Fracture Risk in DMD
Quantifying the Hidden Cost Burden of Duchenne Muscular Dystrophy on U.S. Households
Quantifying the Hidden Cost Burden of Duchenne Muscular Dystrophy on U.S. Households
NeuroFrontiers
Quantifying the Hidden Cost Burden of Duchenne Muscular Dystrophy on U.S. Households
Cardiopulmonary Exercise Testing Sheds Light on Early Physiologic Decline in Duchenne Muscular Dystrophy
Cardiopulmonary Exercise Testing Sheds Light on Early Physiologic Decline in Duchenne Muscular Dystrophy
NeuroFrontiers
Cardiopulmonary Exercise Testing Sheds Light on Early Physiologic Decline in Duchenne Muscular Dystrophy

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