30 closest matches · ranked by relevance
Hereditary Spastic Paraplegia · Adrenomyeloneuropathy
Primary: Change From Baseline Walking Time in 10 Meter Walk Test to the Measurement Taken Directly After rTMS — -5.41; 1.70 seconds
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Hereditary Haemorrhagic Telangiectasia
Primary: HHT Patients Who by the Questionnaire Did or Did Not Self Report a Medical Complication During Flight — 77 percentage of patients
Huntington Disease
Primary: Number of Patients With at Least One Adverse Event — 119 Participants
Muscular Atrophy, Spinal
Primary: Efficacy: Functional Motor Testing, Including Gross Motor Function Measure (GMFM) and Timed Motor Tests
Spasticity
Primary: Least Square Mean Difference (Placebo Versus Each SPARC1103 Dose) in Change From Baseline in Modified Ashworth Score — 0.33; 0.03 score on a scale
HTLV I Associated Myelopathy
Primary: Number of Patient With Lack of Objective Clinical Improvement — 0 Participants
Muscle Spasticity
Primary: Physician Assessment of Spasticity
Albinism · Inborn Errors of Metabolism · Oculocutaneous Albinism
Primary: Change in Forced Vital Capacity (36 Months) — -23.52; -20.93 % of predicted volume
Progressive Supranuclear Palsy · Dementia With Lewy Bodies · Multiple System Atrophy
Primary: Change in Patient Quality of Life as Measured by the EuroQol 5-D (EQ-5D) Scale Between Baseline (Visit 1) and 1 Year (Visit 4) — 1; 0.5; 0.5; 0 units on a scale — p=0.59
Secondary Progressive Multiple Sclerosis · Primary Progressive Multiple Sclerosis
Primary: Number of Participants Classified as Upper Limb Responders on the 9 Hole Peg Test (9HPT) — 4; 3; 58; 59 Participants
Hypophosphatasia (HPP)
Primary: Survival — 270.5 days
Muscular Atrophy, Spinal
Primary: Safety: Frequency of Adverse Events/Lab Abnormalities
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
Spinocerebellar Ataxias · Tai Chi
Primary: Scale for the Assessment and Rating of Ataxia(SARA) — 11.5; 9 score on a scale
Cerebral Palsy, Spastic
Primary: Change in Muscle Size Parameter — 3.7; 1.6; 1.4; 0.1 milliliters — p=<0.01
Huntington Disease
Primary: Safety and Tolerability of PBT2 in Patients With HD — 32; 30; 28 participants
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
Spinal Muscular Atrophy Type 3 · Spinal Muscular Atrophy Type 4
Primary: Muscle Weakness — 2; 3 Participants
Spinocerebellar Ataxia
Primary: Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA) — -25.9; -27.8 percentage change — p=0.29
HIV · Sensory Neuropathy · Neuropathic Pain
Primary: Neuropathic Element of Pain Using the Doleur Neuropathique 4 Interview — 81; 67 Participants
Multiple Sclerosis
Primary: Impact of Spasticity, Measured by the Multiple Sclerosis Spasticity Scale-88 — -16.11; -15.83 units on a scale
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Hyperkalemic Periodic Paralysis · Hypokalemic Periodic Paralysis
Primary: HYP Attack Rate — 0.9; 4.8 attacks per week
Spinal Cord Injury
Primary: Muscle Mass Via Dual-Energy X-ray Absortiometry (DEXA)Scan Data — 6104.45; 6355.3 grams — p=.318
Stroke · Shoulder Pain
Primary: Brief Pain Inventory (BPI)- Short Form (SF) Question 3 (BPI-SF3) - Mean Change From Baseline — 7.1; 7.4; 8.1; -2.8 units on a scale
Cervical Spondylotic Myelopathy
Primary: Virtual Environment TBI System (VETS) Assessment of Static Posturography — 11; 13.0; 48.4; 19.1 Square Centimeters
Peripheral Artery Disease
Primary: Six-minute Walk Distance — 17.01; 3.46 meters — p=0.7586
Spinal Cord Injury at C4-C6 Level
Primary: Change in Voluntary Movement — 2; 0 Participants
Severe Spasticity
Primary: Change in Average Ashworth Scale (AS) in Affected Lower Extremities From Baseline to Month 6 — -0.99; -0.43 units on a scale — p=0.0140