Rare Diseases
Conference Coverage
Combination therapy shows mixed results for scleroderma-related lung disease
A study of mycophenolate mofetil alone and in combination with pirfenidone for scleroderma-related interstitial lung disease showed mixed results...
Conference Coverage
Clinical signs differ between children and adults with vasculitis
Many pediatric rheumatologists believe kids with vasculitis are like small adults and should be treated similarly, but Dr. Jessica Bloom said the...
Guidelines
First recommendations for cancer screening in myositis issued
The guideline, based on data from a systematic review and meta-analysis and on consensus from an international team of experts and patients,...
From the Journals
Gene ‘cut-and-paste’ treatment could offer hope for inherited immune system diseases
“Collecting the T cells is easier and correcting the T cells is easier. With this approach the amount of time in hospital the patients would need...
From the Journals
Goodbye ‘diabetes insipidus’, hello ‘AVP-D’ and ‘AVP-R’
Confusion with the term ‘diabetes mellitus’ ... has spurred proposed name change by an international panel representing major endocrinology...
News
Staying alert for patients with narcolepsy
The chronic neurologic disorder entails not only excessive sleepiness but also social and professional challenges.
News
Novel gene-based therapies for neuromuscular diseases
Novel gene-based therapies show significant potential for transforming the treatment of neuromuscular diseases.
News
Rare disease patient advocacy groups empowered by data
On the IAMRARE platform, patient advocacy organizations are trained to conduct observational research and host natural history.
News
NORD Rare Disease Centers of Excellence: A new network seeks to break down barriers in rare disease care
“The goal was to find places that could help with unanswered questions, whether diagnostic questions or treatment questions.”
News
The broad and challenging – but promising – landscape of peripheral neuropathy
This review of peripheral neuropathy summarizes the more common causative entities, diagnostic tools, and treatments to prevent, slow, or reverse...
News
Myasthenia gravis: Finding strength in treatment options
Although treatment might have once been considered stagnant, newer expert consensus and novel research are generating optimism for innovative...