These data suggest that rhLAMAN may be an encouraging new treatment for patients with alpha-mannosidosis.The study is designed to continue for a total of 18 months. Longer-term follow-up of patients in this study and the future placebo-controlled phase 3 trial are needed to provide greater support for the findings in this study.

Background and Objectives Migraine with aura (MwA) is associated with increased brain hyper‐responsiveness to visual stimuli and increased visual network connectivity relative to migraine without aura (MwoA). Despite this, prior studies have provided conflicting results regarding whether MwA is associated with higher photophobia symptom scores compared to MwoA. The relationships between MwA and other types of sensory hypersensitivity, such as phonophobia and cutaneous allodynia (CA), have not been previously investigated. The purpose of this cross‐sectional observational study was to investigate whether MwA is associated with greater symptoms of photophobia, phonophobia, and CA compared to MwoA. Methods This analysis included 321 migraine patients (146 MwA; 175 MwoA) who had been enrolled into the American Registry for Migraine Research. The diagnosis of either MwoA or MwA was determined by headache specialists using ICHD diagnostic criteria. Patients completed the Photosensitivity Assessment Questionnaire, the Hyperacusis Questionnaire, and the Allodynia Symptom Checklist. Mean or median values were compared between groups. Regression models were created to analyze the relationship between MwA with photophobia scores, hyperacusis scores, and the presence of interictal CA. Results Those with MwA had higher mean photophobia scores than those with MwoA (4.1 vs 3.0, P = .0003). MwA was positively associated with photophobia symptom severity (B = 0.50 [SE = 0.14], P = .0003), after controlling for age, patient sex, and headache frequency. Aura was not associated with hyperacusis symptom severity (B = 0.07 [SE = 0.08], P = .346) or the presence of interictal CA (OR 1.33 [95% CI 0.70‐2.53], P = .381). Conclusion MwA is associated with higher photophobia symptom scores compared to MwoA. Aura is not associated with greater hyperacusis or interictal allodynia scores. These findings complement prior imaging and neurophysiologic studies that demonstrated MwA to be associated with hyper‐responsiveness of brain visual processing regions. The findings suggest that MwA is associated specifically with visual hypersensitivity, as opposed to being associated with a general hypersensitivity to multiple types of sensory stimuli.

Background and Objectives.-The association between migraine, depression, and anxiety has been established, but the impact of these psychiatric comorbidities on functional impairment in people with migraine has been under-investigated. The purpose of this cross-sectional observational study was to investigate the relationship between anxiety and depression symptoms on migraine-related disability, pain interference, work interference, and career success in a cohort of patients with migraine. Methods.-This analysis included 567 migraine patients who had been enrolled into the American Registry for Migraine Research (ARMR) between February 2016 and June 2019. Patients completed the Generalized Anxiety Disorder-7 (GAD-7) and Patient Health Questionnaire-2 (PHQ-2) to measure symptoms of anxiety and depression, respectively. Patients completed the Migraine Disability Assessment Scale (MIDAS), Pain Interference (PROMIS pain short) questionnaire, and Work Productivity and Activity Interference (WPAI) questionnaire to measure levels of functional impairment at work and in daily activities. In addition, patients answered questions designed for ARMR regarding education and career interference. Models were created to describe the relationship between severity of psychiatric symptoms (anxiety and depression), and each outcome of interest (WPAI, MIDAS, pain interference, reporting that migraine had interfered with career success). Each model was controlled for age, sex, headache frequency, years with migraine, and average headache intensity. Results.-Among the 567 patients with migraine, mean (SD) age was 47.1 (13.7), 87.3% were female, and average headache frequency was 19.1 (9.3) days/month. PHQ-2 scores were positively associated with scores on MIDAS (b = 0.

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Objectives Improving health-related quality of life (HRQoL) is a major treatment goal for inflammatory bowel disease (IBD) patients. Tools to measure HRQoL, including the Pittsburgh Sleep Quality Index and the Short Inflammatory Bowel Diseases Questionnaire, are lengthy and rely on recall. This prospective, proof-of-concept pilot study assessed the feasibility, reliability, and validity of daily, simplified HRQoL and sleep quality data collection using mobile technologies in patients with IBD. Methods Adult IBD patients were recruited from our center. Daily HRQoL and sleep quality were assessed using visual analog scale (VAS) surveys, and pain was assessed using the Wong-Baker FACES Pain Rating Scale (WBS). HRQoL and sleep were assessed on days 1, 14, and 28 of the study using the short IBD Questionnaire (SIBDQ) and Pittsburgh Sleep Quality Index (PSQI). Correlation between the daily instruments and the biweekly validated instruments were assessed using the Pearson correlation coefficient. Results One hundred patients were enrolled. The correlation between mean global assessment VAS score over 2 weeks and PSQI score taken at 14 days was moderate (r = 0.62, P < 0.0001). The correlation over the same time interval between mean WBS score and SIBDQ score was strong (r = −0.71, P < 0.0001), and the correlation between mean sleep VAS score and PSQI score was moderate (r = −0.55, P < 0.0001). Conclusions This study demonstrates the potential for electronic quality of life, sleep quality, and pain assessments as feasible, reliable, and valid tools in IBD patients. Intermittent administration of these simplified electronic assessments may be useful in further reducing patient survey burden without significantly compromising their utility.

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