Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Must Reads In Rare Diseases

Rare Diseases for September/October 2019

Neurologists publish consensus statement on stridor in MSA,

Cerliponase Alfa Impresses for CLN2 Disease,

More must reads

Rare Diseases for July/August 2019

ALS Risk Elevated Among First-Degree Relatives of Patients With Disease,

Mutant Huntingtin and Neurofilament Light Valid Biomarkers in Huntington’s Disease,

Genetic Therapy Reduces Key Protein in Huntington’s Disease,

More must reads

Rare Diseases for May/June 2019

Inebilizumab Reduces Risk of NMOSD Attacks,

Can an Antisense Oligonucleotide Benefit Patients with SOD1-ALS?,

Is Professional Soccer a Risk Factor for ALS?,

Angelman Syndrome Treatment Safe, Effective in Exploratory Analyses,

More must reads

Rare Diseases for March/April 2019

Polyglutamine Diseases Are Rare, But Not Mutations That Cause Them,

Trofinetide May Benefit Patients with Rett Syndrome ,

More must reads

Rare Diseases for November/December 2018

FDA Approves Firdapse for Rare Autoimmune Disorder, FDA news release; 2018 Nov 28
More must reads

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