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Seeking Breakthroughs—We Want You!

Shamik Bhattycharyya, MD, MS

September 2018—The challenges we must overcome to find breakthrough treatments in neurology are many and the potential is great.

Nusinersen Effective Treatment for Type 1 Spinal Muscle Atrophy Even When Given at Age 7 Months and Up

Wednesday, August 29, 2018—Nusinersen (Spinraza; Biogen, Cambridge, MA), a novel therapy approved by the Food and Drug Administration (FDA) for treatment of patients with spinal muscle atrophy (SMA). Re…

First-in-Class RNAi Treatment for Hereditary Transthyretin-Mediated Amyloidosis

Wednesday, August 15, 2018—The US Food and Drug Administration (FDA) has approved patisiran (Onpattro; Alnylam Pharmaceuticals, Cambridge, MA) for treatment of polyneuropathy in adults with hereditary t…

Breaching the Blood-Brain Barrier: A Potential New Treatment Modality

Monday, July 30, 2018—In a small, yet groundbreaking, clinical trial, MR-guided focused ultrasound (MRgFUS) (ExablateNeuro; Insightec, Miami FL) was used to open the blood-brain barrier (BBB) safel…

Amyotrophic Lateral Sclerosis the Next Target for Antisense Oligonucleotide Therapy?

Thursday, July 26, 2018—Antisense oligonucleotide (ASO) therapy is a new class of treatment of people with illnesses caused by defects in a single gene. In this novel approach, instead of repairing o…

IncobotulinumtoxinA Approved for Treatment of Excessive Drooling

Friday, July 13, 2018—The Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for incobotulinumtoxin A (Xeomin; Merz, Raleigh, NC) for the treatmen…

BrainStorm Will not Make Investigational Nurown Stem Cell Treatment Available Under Right-to-Try Legislation

Tuesday, June 26, 2018—BrainStorm Cell Therapeutics Inc. has announced that after careful consideration of the newly enacted right-to-try (RTT) legislation, it will not make stem-cell therapy (Nurow…

Recurrent Stroke-Like Events Before Diagnosis of X-Linked Charcot—Marie—Tooth Disease

Arayamparambil C. Anilkumar, MD

June 2018—Awareness of pathognomonic findings may prevent unnecessary tests and treatments.

 
 

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About Practical Neurology

Launched in 2002, Practical Neurology is a publication uniquely dedicated to presenting current approaches to patient management, synthesis of emerging research and data, and analysis of industry news with a goal to facilitate practical application and improved clinical practice for all neurologists. Our straightforward articles give neurologists tools they can immediately put into practice.

 
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