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Optimizing Drug Selection in Children with Multiple Sclerosis: What Do We Know and What Remains Unanswered?

Paediatr Drugs

December 2024

Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, 555 University Avenue, Toronto, ON, M5G 1X8, Canada.

Pediatric-onset multiple sclerosis (POMS) refers to multiple sclerosis with onset before 18 years of age. It is characterized by a more inflammatory course, more frequent clinical relapses, and a greater number of magnetic resonance imaging (MRI) lesions compared with adult-onset MS (AOMS), leading to significant impacts on both disability progression and cognitive outcomes in affected individuals. Managing POMS presents distinct challenges due to the unique needs of pediatric patients and the limited number of disease-modifying therapies (DMTs) approved for pediatric use.

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Article Synopsis
  • SARS-CoV-2, the virus that caused COVID-19, led to a global pandemic with many cases and deaths, prompting scientists to quickly create vaccines.
  • The review looked at different COVID-19 vaccines, their benefits, side effects, and how they might affect people with multiple sclerosis (MS), especially those on specific medications.
  • Overall, getting vaccinated against COVID-19 is much safer than getting sick from the virus, and the advantages of the vaccine for people with MS are greater than the risks, although doctors should watch for complications.
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Tofersen for SOD1 ALS.

Neurodegener Dis Manag

October 2024

Department of Neurology, Washington University School of Medicine, Saint Louis, MO 63110, USA.

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  • Amyotrophic lateral sclerosis (ALS) is a complex neurological disease that impacts muscle control, making research and clinical trials challenging due to its varied nature.
  • Genetic mutations causing ALS, particularly those involving SOD1, allow for targeted treatment strategies, such as the 'knock-down' approach that reduces harmful protein production.
  • Tofersen, an experimental treatment that decreases SOD1 mRNA, showed promising biomarker results, and although its Phase III trial didn’t hit its main goal, follow-up data indicate it may help slow down the progression of the disease in SOD1 ALS patients.
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Multiple sclerosis (MS) is a chronic, demyelinating disease affecting the central nervous system. Diagnosis of MS is based on the proof of disease dissemination in time (DIT) and dissemination in space (DIS) and excluding other disorders that can mimic multiple sclerosis in laboratory tests and clinical manifestation. Over the years the diagnostic criteria have evolved; the introduction of magnetic resonance in the McDonald's 2001 criteria was revolutionary.

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