Rare Diseases
Conference Coverage
Novel myasthenia gravis therapies bring opportunities, challenges
Minimal disease manifestation is meaningful for patients, but should be weighed against potential side effects.
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...
Supplement
Spotlight on SMA, Part 2: The Spinal Muscular Atrophy Treatment Landscape
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.
Conference Coverage
Duchenne muscular dystrophy gene therapy safe, effective at 4 years
“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...
Conference Coverage
In myasthenia gravis, antibodies pass open-label tests
New class of therapies interfere with IgG recycling and have similar efficacy across multiple cycles of treatment.
FDA/CDC
FDA okays drug for Duchenne muscular dystrophy
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
A new chapter for research on treating Huntington’s disease
BY JENNIE SMITH
The past decade has been a contradictory one for research on Huntington’s disease, marked by breakthroughs in the biology...