Duchenne muscular dystrophy: Drug development and regulatory considerations.

Duchenne muscular dystrophy (DMD) is one of the most commonly recognized dystrophinopathies. There are no approved therapeutic options available for this disease but recent discoveries have led to hope that effective therapies might be forthcoming. With funding from patient advocacy groups, private investors, and governmental bodies such as the Food and Drug Administration Office of Orphan Product Development (FDA/OOPD), gene modification and other molecular therapies are being actively investigated.

An evaluation of a data mining signal for amyotrophic lateral sclerosis and statins detected in FDA's spontaneous adverse event reporting system.

Background: We detected disproportionate reporting of amyotrophic lateral sclerosis (ALS) with HMG-CoA-reductase inhibitors (statins) in the Food and Drug Administration's (FDA) spontaneous adverse event (AE) reporting system (AERS).

Purpose: To describe the original ALS signal and to provide additional context for interpreting the signal by conducting retrospective analyses of data from long-term, placebo-controlled clinical trials of statins.

Methods: The ALS signal was detected using the multi-item gamma Poisson shrinker (MGPS) algorithm.

Revised statistical motor unit number estimation in the Celecoxib/ALS trial.

Techniques to estimate motor unit number (MUNE) measure the number of functioning motor units in a muscle. As amyotrophic lateral sclerosis (ALS) is characterized by progressive motor unit loss, this disease offers an ideal setting for the use of MUNE. Statistical MUNE was employed in a recent multicenter trial of creatine in ALS, and was shown to be reliable, reproducible, and to decline with disease progression.

Electrodiagnosis in common mononeuropathies and plexopathies.

Electrodiagnosis has a prominent role in the diagnosis of common entrapment neuropathies (carpal tunnel syndrome and ulnar neuropathy at the elbow) and plexopathies (idiopathic brachial plexopathy and diabetic lumbar radiculoplexopathy). The relevant anatomy and pathology of these disorders is reviewed in the context of electrodiagnosis and prognosis.