Minimal disease manifestation is meaningful for patients, but should be weighed against potential side effects.
“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
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...
A study of the NIH All of Us database shows a higher prevalence of myasthenia gravis than what has been reported in previous studies.
“We now have the potential to change the way genetic epilepsies are treated by addressing the underlying genetic cause of the disease instead of...
Long waits for neuropsychiatric evaluations make it difficult to get help, but an NIH tool may help.
A new subcutaneous injectable showed efficacy and tolerability in patients with chronic inflammatory demyelinating polyneuropathy.
“What’s really striking, and in my mind the most impressive, is that when you follow these patients out 3 or 4 years ... you see there is this...
New class of therapies interfere with IgG recycling and have similar efficacy across multiple cycles of treatment.
The approval of vamorolone “provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some...
Rare Diseases Report 2023
BY JENNIE SMITH
The past decade has been a contradictory one for research on Huntington’s disease, marked by breakthroughs in the biology...