Rare Diseases
Article
FDA Launches Expanded Access Navigator
Article
Examining the Role of Caregivers at NINR Caregiving Summit
Article
FDA Commissioner to Present Keynote Address at NORD Summit
Article
Preview Agenda for NORD’s 2017 Rare Diseases and Orphan Products Breakthrough Summit
Conference Coverage
Screening MRI misses Sturge-Weber in babies with port-wine stain
In a small study of children with high-risk port-wine stains, MRI screening for Sturge-Weber syndrome had a false-negative rate of 23%.
Conference Coverage
Care of infants with ichthyosis requires ‘all hands on deck’
The neonatal period and early infancy are especially critical for patients with ichthyosis, because compromised barrier function increases risk...
Conference Coverage
Precise cause of pityriasis rosea remains elusive
Pityriasis rosea was recognized as early as 1798, yet its precise cause remains elusive.
Commentary
Adalimumab for Hidradenitis Suppurativa
We applaud Kimball et al1 on their report that adalimumab demonstrated clinical improvement in patients with hidradenitis suppurativa (...
Conference Coverage
Bone marrow transplantation for epidermolysis bullosa continues to evolve
Today, bone marrow transplantation is the most widely used stem cell therapy for patients with severe EB.
Conference Coverage
Many aspects to caring for epidermolysis bullosa patients
CHICAGO – An expert describes treatment options for the symptoms considered most bothersome by patients with recessive dystrophic epidermolysis...
Conference Coverage
Supportive care alone linked to worse outcomes in mucocutaneous reactions
CHICAGO – Supportive care and surgical debridement may not be enough when treating pediatric patients with SJS/TEN.