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More Rare Diseases News
- FDA approves Givlaari for treatment of acute hepatic porphyria
- AVXS-101 may result in long-term motor improvements in SMA
- Ataluren shows real-world benefit for nonsense mutation Duchenne muscular dystrophy
- Families face challenges of gene therapy
- CMT1A neuropathy improves with investigational drug PXT3003
This Just In
- Positive functional results reported for aducanumab in a pooled, post hoc analysis
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- Oral contraceptive use associated with smaller hypothalamic and pituitary volumes
- Thanksgiving took a bite out of HealthCare.gov
- Intensive BP control reduced dementia but increased brain atrophy and hurt cognition
Conference Coverage
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Headache may be a significant outcome of pediatric hemispherectomy -
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Late-onset MS is often more severe than earlier-onset MS -
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Children may develop prolonged headache after concussion
Clinical Edge
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AVXS-101 May Result in Long-Term Motor Improvements in SMA
November 18, 2019
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CMT1A Neuropathy Improves With Investigational Drug PXT3003
November 12, 2019
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Ataluren shows real-world benefit for nonsense mutation Duchenne muscular dystrophy
November 7, 2019
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Neurologists publish consensus statement on stridor in MSA
October 15, 2019
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Cerliponase Alfa Impresses for CLN2 Disease
September 17, 2019
