Rare Disease Day: taking a closer look at rare neurological diseases
To mark Rare Disease Day (28 February 2021), we’ve compiled a list of recent articles from our partnered journals that span across neuroscience and neurology. From personal insights into early-onset Alzheimer’s disease, to advancements made in small cell glioblastoma multiforme, take a look at what’s new in the field of rare neurological diseases below.
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Neurodegenerative Disease Management
- Clinical characteristics of Alexander disease
- Riluzole for the treatment of amyotrophic lateral sclerosis
- Please remember the real me when I cannot remember you – experiences with early-onset Alzheimer’s disease
- Long-term normalization of cognitive and psychopathological alterations in a juvenile Niemann–Pick type C case
- Experience of patisiran with transthyretin stabilizers in patients with hereditary transthyretin-mediated amyloidosis
- Efficacy and safety of mexiletine in amyotrophic lateral sclerosis: a systematic review of randomized controlled trials
- Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
- Satralizumab in the treatment of neuromyelitis optica spectrum disorder
- Omaveloxolone: potential new agent for Friedreich ataxia
CNS Oncology
- A peek behind the paper – Katy Peters on ibrutinib in primary central nervous system diffuse large B-cell lymphoma
- Small cell glioblastoma multiforme: a case series and clinicopathological update
- Intermixed arteriovenous malformation and hemangioblastoma: case report and literature review
- Adult pilocytic astrocytoma in the molecular era: a comprehensive review
Future Oncology
- An overview of meningiomas
- Brain tumors in Li-Fraumeni syndrome: a commentary and a case of a gliosarcoma patient
- Pediatric ependymoma: current treatment and newer therapeutic insights
- Evolving molecular era of childhood medulloblastoma: time to revisit therapy
- Advances in the management of brain metastases from cancer of unknown primary
Journal of Comparative Effectiveness Research
- Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Future Drug Discovery
- On the development of miransertib for rare diseases: an interview with ArQule’s Brian Schwartz
- The current state of drug repurposing and rare diseases: an interview with Paul Trippier
Future Neurology