Rare Diseases
Conference Coverage
Essential Tremor Tied to a Threefold Increased Risk for Dementia
During the follow-up, the cumulative prevalence of dementia was 18.5%, and the average annual conversion rate of MCI to dementia was 12.2% —...
Literature Review
Promising Results for Investigational Myasthenia Gravis Drug
After 6 weeks of treatment, patients reported nearly 60% sustained improvement in daily activities.
From the Journals
5 Interesting Neurology Studies
Commentary
Why Are Prion Diseases on the Rise?
Given these findings, it seems that a bit more surveillance for this rare but devastating condition is well merited.
Conference Coverage
Novel myasthenia gravis therapies bring opportunities, challenges
Minimal disease manifestation is meaningful for patients, but should be weighed against potential side effects.
Supplement
Spotlight on SMA, Part 2: The Spinal Muscular Atrophy Treatment Landscape
Conference Coverage
Newly approved myasthenia gravis drug shows sustained benefits in early responders
“This self-administered medication allows patients to be more independent and can even travel since it is not dependent on an infusion center.”
From the Journals
Short steroid taper tested with tocilizumab for giant cell arteritis
Results from a pilot study examining a shortened prednisone taper of 8 weeks’ length rather than 26 weeks suggest it may be possible to reduce...
Conference Coverage
U.S. study finds unexpectedly high prevalence of myasthenia gravis
A study of the NIH All of Us database shows a higher prevalence of myasthenia gravis than what has been reported in previous studies.
Literature Review
RNA therapeutics will ‘change everything’ in epilepsy
“We now have the potential to change the way genetic epilepsies are treated by addressing the underlying genetic cause of the disease instead of...
Conference Coverage
‘Hidden’ cognitive impairments in DMD may worsen outcomes
Long waits for neuropsychiatric evaluations make it difficult to get help, but an NIH tool may help.
Conference Coverage
First new treatment in 30 years for rare disease is effective, tolerable, convenient
A new subcutaneous injectable showed efficacy and tolerability in patients with chronic inflammatory demyelinating polyneuropathy.