Rare Diseases
Latest News
New data confirm risk of Guillain-Barré with J&J COVID shot
Nearly all individuals who developed GBS after the Janssen vaccine had facial weakness or paralysis, in addition to weakness and decreased...
Latest News
A gruesome murder changes two docs’ lives, and one was the killer
“Four years later, his patients were still shocked about what happened and couldn’t reconcile the person they knew with the event that happened...
Conference Coverage
Steroids counter ataxia telangiectasia
Red blood cells infused with dexamethasone sodium phosphate delayed symptom progression and improved survival.
From the Journals
Restless legs syndrome occurs often in X-linked adrenoleukodystrophy
Patients with restless legs syndrome showed less brain demyelination than those without RLS.
Conference Coverage
Nanoparticle shows promise for ALS
A phase 2 study revealed a significant survival advantage with drug that targets mitochondria.
Conference Coverage
Brain implant is a potential life-changer for paralyzed patients
The minimally invasive device enables patients to carry out important activities of daily living.
Conference Coverage
Novel drug significantly reduces tics in Tourette syndrome – without side effects
Ecocipam does not lead to weight gain, anxiety, depression, or tardive dyskinesia, compared with placebo – a factor that may lead to better...
Conference Coverage
Gene therapy demonstrates modest success in genetic blindness
‘First step’ toward improvement in Leber hereditary optic neuropathy is maintained out to 4 years.
FDA/CDC
FDA advisory panel rejects new ALS drug
The FDA panel raised a number of issues with the clinical trial, including the study’s small sample size and no survival benefit at 24 weeks.
From the Journals
Surgery in CJD patients a potential risk factor for transmission
Literature Review
Brain implant allows fully paralyzed patient to communicate
The study shows for the first time that communication is possible in patients in a completely locked-in state and offers hope for a better quality...