Nerve Injury

4-Aminopyridine

Placebo oral tablet

Hand sensibility, strength, and laxity of high-level musicians compared to nonmusicians

A Single Dose Pharmaco-Diagnostic for Peripheral Nerve Continuity After Trauma

Return of lost sensation after nerve injury attributable to circulating 4-AP. Objective return of voluntary function, motion in the injured limb or portion of the limb in one minute of testing. The descriptive name of the scale is "volitional motion of the injured limb in one minute".
Patients for this trial are not able to move or sense in portions of their limbs. The measure will be motion, measured on the binary scale (able to initiate voluntary motion vs unable to initiate voluntary motion). This is assessed through clinical examination of the injured limb. The volitional control of motion is widely accepted as a correlate to nerve function. Expressed in terms of rate, it would be the number of times, in one minute that a patient can move a limb or portion of a limb, that they were unable to move before the trial.

The results of this study include both subjective and objective measures. Subjective measures will not be hidden from patients given the nature of measurements. Active discussion of objective measures will wait until final follow up at 20 weeks. Patients can use their subjective assessments in their standard-of-care treatment decisions.Investigators will not inform other physicians except at the consent of patients. Subjective responses to treatment can be discussed by patients with treating physicians. Objective test results will not be discussed with treating physicians until 20 weeks. If patient wants further surgical exploration of an injured peripheral nerve based on subjective return of function (or lack) with members of the study team, a patient conference will be arranged with ethicist and DSMB. The patient's decision in conjunction with the treating surgeon (not on study team to avoid any conflicts) is to be paramount. Unblinding will also be discussed then with the DSMB.

John Elfar

Michael and Myrtle Baker Professor and Vice Chair of Research, Department of Orthopaedics and Rehabilitation

Milton S. Hershey Medical Center

Miglustat is administered, dose is adjusted according to Body Surface Area as below:
>1.25 : 200 mg TDS 0.88-1.25 : 200mg BID 0.73-0.88 :100mg TDS 0.47-0.73 : 100mg BID <0.47 :100mg daily

GM2 Gangliosidosis

Supportive Care

Treatment with Zavesca regimen based on body surface area as follows: SQRT [Height (cm) × Weight (kg)] / 3600 <1.25 : 200mg TDS 0.88- 1.25: 200mg BID 0.73- 0.88: 100 mg TDS 0.47- 0.73: 100 mg BID <0.47: 100 mg Daily

Miglustat

Kashan University Of Medical Sciences

Mashhad University Of Medical Sciences

Tehran University Of Medical Sciences

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Survey of Miglustat Therapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sandhoff and Taysachs Diseases

A total score is reported according to Pediatric Quality Of Life Inventory Infant Scales. Total score range is between 0-45 and higher values represent worse outcomes.

Tavasoli

Principal investigator

Tehran University of Medical Sciences

Mashhad University of Medical Sciences

Kashan University of Medical Sciences

Although randomized control trial is the gold standard for clinical trial studies; there are ethical concerns about placebo control group in rare diseases such as Sandhoff and Tay sachs diseases.

muscular atrophy/эпилептический припадок

A Single Dose Pharmaco-Diagnostic for Peripheral Nerve Continuity After Trauma

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Самая полная база данных о лекарственных травах, подтвержденная наукой