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Stem Cell Transplantation for MS Treatment

In this small study of the effect of mesenchymal stem cell treatment, both intravenous and intrathecal, in patients with progressive multiple sclerosis, the treatment was shown to be safe, and there was a trend showing some benefit in slowing disease progression, compared to the sham group. A larger randomized controlled trial is needed to confirm these early findings.

Spontaneous Intracranial Hypotension Syndrome

A systematic review of spontaneous intracranial hypotension demonstrates heterogeneity in clinical and radiographic presentations. Diagnostic studies, such as brain imaging and lumbar puncture, may be unrevealing, and the clinician may have to rely on symptom patterns alone to make a diagnosis.

Efficacy and Safety of Cannabidiol in Treatment of Focal Seizures Associated with Tuberous Sclerosis Complex

Cannabidiol used as an add-on therapy for intractable focal seizures in patients with tuberous sclerosis complex was efficacious. Cannabidiol also had an acceptable adverse event profile, but frequently induced transient elevation of liver enzymes.

Gluten/Celiac Disease Neuropathy

Gluten neuropathy should be suspected in any patient who presents with a neuropathy and has a history of gastrointestinal disease. The diagnosis is made using blood serology plus intestinal mucosal biopsy.

Long-Term Treatment of Hereditary Amyloid Neuropathy with Patisiran

Hereditary transthyretin-mediated amyloidosis with polyneuropathy is effectively treated by patisiran, a lipid nanoparticle ribonucleic acid interference that binds to transthyretin (TTR) messenger RNA and inhibits TTR production in the liver, resulting in reduction of the serum TTR concentration. Treatment effect has been sustained during a five-year open-label extension study.

Clinical and Genetic Characterizations to Diagnose Sarcoglycanopathies

Sarcoglycanopathies, caused by various genetic mutations, may cause limb-girdle forms of muscular dystrophy early in life. Although there are no specific treatments for these disorders at this time, the clinical and genetic characterizations will assist in more precise diagnosis that will be critical to develop new molecular-based therapies.

Prednisone vs. Placebo in Short-Term Prevention of Episodic Cluster Headaches

Prednisone, given at 100 mg for five days and then tapering by 20 mg every three days, is a safe and effective short-term prevention for episodic cluster headaches while waiting for longer-acting preventive agents to be initiated.

Cognitive Benefit of Rivastigmine in Parkinson’s Disease Dementia with Orthostatic Hypotension

Individuals with Parkinson’s disease dementia and orthostatic hypotension (OH) have more robust cognitive improvement from rivastigmine compared to those without OH. This greater response possibly is mediated by the anti-OH effect of rivastigmine.

Deficits in Neurotransmitters and Behavioral Disturbances in Frontotemporal Lobar Degeneration

In subjects with a syndrome associated with frontotemporal lobar degeneration, gamma aminobutyric acid and glutamate deficiency in the right inferior frontal gyrus was associated with greater degrees of impulsivity.

Focused Ultrasound in Parkinson’s Disease: Going Beyond Tremor

In this randomized controlled trial, patients with asymmetric Parkinson’s disease were assigned to noninvasive, focused ultrasound-mediated subthalamotomy or sham procedure. The authors reported improved motor function in the focused ultrasound group, with adverse events including weakness, dyskinesia, and gait disturbances.