FDA Approves Dysport for Upper Limb Spasticity in Children

The U.S. Food and Drug Administration (FDA) has expanded the use Dysport (botulinum toxin type A) to include the treatment of upper limb spasticity in children two years of age and older, excluding spasticity caused by cerebral palsy. The FDA previously approved...

FDA Approved Vaccine for Smallpox and Rare Monkeypox Disease

The U.S. Food and Drug Administration (FDA) has approved Jynneos, a vaccine designed to prevent smallpox and monkeypox disease. Smallpox afflicted thousands in the early part of the 20th century but was eradicated in the 1980s following an aggressive vaccination...

Autism Versus Phelan-McDermid Syndrome

  Phelan-McDermid syndrome is a rare genetic condition due to mutations in chromosome 22. In most cases, the mutations are de novo and the variety of mutations leads to a large array of symptoms and their severity. A common set of symptoms however, are...

Jansen’s Disease Research Update

  Jansen’s metaphyseal chondrodysplasia is an extremely very rare autosomal dominant disease. It is a type of skeletal dysplasia characterized by short-limbed short stature, waddling gait, bowed legs, contracture deformities of the joints, short hands,...

Fibromuscular Dysplasia (FMD) Overview

  Jeffrey W Olin, DO is a Professor of Medicine at the Icahn School of Medicine at Mount Sinai and Director of Vascular Medicine and the Vascular Diagnostic Laboratory in the Zena and Michael A. Wiener Cardiovascular Institute and Marie-Josée and Henry R. Kravis...

Approved and Emerging Therapies for Mucopolysaccharidoses (MPS)

  Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the approved and emerging therapies for mucopolysaccharidoses...
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