The current behavioral intervention consists of two primary components; an internet website and oneonone video chats with a behavioral coach.The internet website involves content delivered through interactive video courses.The interactive video courses are based on elements of social cognitive theory.Each course consists of an introduction,the primary content,and a take home message.The interactive courses include embedded,supplementary options such as videos on content and worksheets related to the topic.A pedometer is provided for tracking steps,and these steps will be entered into the website so progress can be monitored.The chats support adherence to the intervention,discussion of website material,supportive accountability,and reporting of adverse events/injuries.The chats are conducted facetoface through an online videoconferencing platform.The chats occur 7 times during the first 2 months,4 times during the second 2 months,and twice during the final 2 months of the intervention.

A behavioral intervention that involves an internet website and one-on-one video coaching calls for increasing physical activity in people with MS.

Behavioral Intervention for Physical Activity in MS (BIPAMS)

Provides an internet website and oneonone video chats that discuss materials about self-managing multiple sclerosis (MS) consequences and health indicators through methods other than physical activity.The materials are transformations of brochures provided by the National MS Society,including Gait or Walking Problems:The Basic Facts;MS and Your Emotions;Pain:The Basic Facts; Solving Cognitive Problems;Taming Stress in MS;Food for Thought:MS and Nutrition;and Vitamins,Minerals,and Herbs:An Introduction.The delivery of the internet materials and chat sessions will occur on the same time schedule and frequency as the intervention condition,and will have a comparable time commitment. The control condition will not involve tracking steps and a pedometer with not be provided.

A behavioral intervention that involves an internet website and one-on-one video coaching calls for increasing wellness in people with MS.

Wellness for MS (WellMS)

Robert W Motl, PhD

The proposed exercise training program is based on Social Cognitive Theory (SCT) for promoting aerobic and strength training in wheelchair users with MS. The 16-week program includes one-on-one video chats with a behavioral coach and various "tracks" for progressive increases in both strength and aerobic training. The proposed strength training includes 15 exercises with step-by-step instructions using resistance bands and/or wrist weights that were adapted for seated body-position that can be executed in a wheelchair. The proposed aerobic training is arm cycle ergometry; this is a low-cost and modifiable option that fits the needs of both power and manual wheelchair users. Other proposed equipment includes a comprehensive Training Manual, fitness tracker for use during exercise training sessions, and Rate of Perceived Exertion (RPE) scale for guiding individualized exercise intensity. The research team created 12 Newsletters that align with 12 proposed coaching calls.

Seated Physical activity INtervention for persons with MS, "SPIN" program- Intervention Condition

The attention/contact wellness control condition will mirror the exercise training intervention condition, but focuses on implementing health behaviors other than physical activity (e.g., diet and emotional wellbeing). This program is an adapted version of the WellMS program from Prof. Motl's Phase III clinical trial (NCT03490240). The program is based on SCT principles of behavior change and integrates wellness resources from the National MS Society (NMSS). The research team has created 12 Newsletters that will be delivered on the same chat schedule as the exercise training intervention. All coaching chats and newsletters will occur with the same frequency as in the exercise training intervention condition. Participants will be provided with a participant manual, logbook, and calendar. Participants will work with their behavioral coach on goal setting specific to wellness behaviors.

WEllness for Longevity Living with MS (WELL)- Control Condition

The University of Texas Health Science Center, Houston

Telehealth visits will be performed using institutionally-approved, secure, web-based teleconferencing. The standard neurology visits will occur every 6 months with their established neurology clinician via telehealth. The comprehensive care will be offered via telehealth or within the patient's local community. The comprehensive care visits will be adapted for the individual participants' needs and symptoms, consistent with standard clinical care.

Intervention includes clinical care delivered via telehealth

Multiple Sclerosis Clinical Care Delivery via telehealth

Standard neurology visits will be conducted in-clinic visits every 6 months with their established neurology clinician. The comprehensive MS care visits will be conducted in-clinic. The comprehensive care visits will be adapted for the individual participants' needs and symptoms, consistent with standard clinical care.

Intervention includes clinical care delivered via standard in clinic visits

Multiple Sclerosis Clinical Care Delivery via standard in clinic visits

MS Participant Group 1 will receive the HOBSCOTCH-MS intervention consisting of 1:1 sessions delivered once per week including:

* 1 pre-HOBSCOTCH
* Session (on webcam)
* 1 educational session (on webcam)
* 6 telephone sessions
* 1 wrap-up session (webcam or telephone)

HOBSCOTCH is a home-based self-management program to treat cognitive symptoms and improve quality of life, while minimizing the barriers of access to care. The program is based on Problem Solving Therapy (PST) and teaches problem solving strategies and compensatory mechanisms to help manage cognitive dysfunction and enhance quality of life. HOBSCOTCH-MS is an adaptation of the HOBSCOTCH program specifically for people who have multiple sclerosis. It incorporates education about these conditions and cognition into the education module.

Home Based Self- management and Cognitive Training Changes lives in Multiple Sclerosis (HOBSCOTCH-MS)

MS Participant Group 2 will be on a 3-month wait list after which they will receive HOBSCOTCH-MS consisting of 1:1 sessions delivered once per week including:

* 1 pre-HOBSCOTCH
* Session (on webcam)
* 1 educational session (on webcam)
* 6 telephone sessions
* 1 wrap-up session (webcam or telephone)

Caregiver Group 1 will receive an adapted version of HOBSCOTCH-MS by attending the introductory session and session 1 of the HOBSCOTCH-MS (virtual) program with their MS participant as well as guidance and instructions on utilizing quick relaxation. Caregiver Group 1 will also attend session 8 of the program with the MS patient to focus on program wrap-up and maintenance planning.

Caregiver Group 2 will be on a 3 month wait list with their MS participant after which time they will receive an adapted version of HOBSCOTCH-MS by attending the introductory session and session 1 of the HOBSCOTCH-MS (virtual) program with their MS patient as well as guidance and instructions on utilizing quick relaxation. Caregiver Group 1 will also attend session 8 of the program with the MS patient to focus on program wrap-up and maintenance planning.

Meredith E Olenec

Sarah J. Kaden, BA

Elaine T. Kirikapoulos, MD, MPH, MSc

This is a prospective, interventional study involving 30 subjects who have been diagnosed with multiple sclerosis and being treated with Ocrelizumab for at least one year.

The cognitive and fatigue assessments administered in this study are not currently part of the standard clinical care for multiple sclerosis patients on Ocrelizumab. By prospectively assigning participants to undergo these additional assessments, this aspect of the study is considered investigational, and findings may contribute to clinical decision-making regarding the incorporation of cognitive and fatigue assessments into routine management of multiple sclerosis conditions.

Multiple Sclerosis patients who have been on Ocrelizumab will undergo cognitive and fatigue assessments

Lynn Huber, MBA

Sara Omar, MSc

Anza Memon, M.D, FAAN

Kyrstina Mariouw, PhD

Participants in this condition will receive a 4-month home-based, remotely supported aerobic and resistance exercise program based on the Guidelines for Exercise in Multiple Sclerosis (GEMS).

Remotely-coached/guided, home-based program delivered using telerehabilitation focusing on aerobic fitness and muscle strength as a mode of training.

Guidelines for Exercise in Multiple Sclerosis (GEMS)

Participants in this condition will receive a 4-month home-based, remotely supported stretching program emphasizing flexibility and range of motion as important components of fitness based on Stretching for People with MS: An Illustrated Manual from the National MS Society.

Remotely-coached/guided, home-based program delivered using telerehabilitation focusing on stretching and range of motion as the mode of training.

FLEX Stretching and Toning Program

Edson Flores, MPH

The current behavioral intervention consists of two primary components; an internet website and one-on-one video chats with a behavioral coach. The internet website involves content delivered through interactive video courses.The interactive video courses are based on elements of social cognitive theory. Each course consists of an introduction, the primary content, and a take home message.The interactive courses include embedded, supplementary options such as videos on content and worksheets related to the topic.A pedometer is provided for tracking steps, and these steps will be entered into the website so progress can be monitored. The chats support adherence to the intervention,discussion of website material,supportive accountability,and reporting of adverse events/injuries. The chats are conducted face-to-face through an online videoconferencing platform. There are a total of 12 chats over 16 weeks. Chats occur on weeks 1-8, 10-11, 13 and16.

Behavioural intervention for physical activity for multiple sclerosis (BIPAMS)

Participants will have 16-weeks of no intervention or interaction.

유전 대사 이상 및 심각한 골경화증 환자에게 줄기세포 이식의 안전성과 효과를 평가하는 2단계 시험입니다.

Inherited Metabolic Disease (IMD) - Except Haplo-Identical

See intervention descriptions.

Stem Cell Transplantation

* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan

IMD Preparative Regimen

Severe Osteoperosis (OP) - Except Haplo-Identical

See intervention descriptions.

* Anti-thymocyte Globulin (ATG)
* Fludarabine
* Busulfan
* Thiotepa

Osteopetrosis Only Preparative Regimen

Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD)

-Haplo-Identical Only

See intervention descriptions.

* Rituximab
* Alemtuzumab
* Busulfan
* Fludarabine

Osteopetrosis Haploidentical Only Preparative Regimen

N-acetylcysteine start day +1 through day +28

cALD SR-A (Standard-Risk, Regimen A)

N-acetylcysteine start day +1through day +56

cALD SR-B (Standard-Risk, Regimen B)

N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100

cALD HR-D (High-Risk, Regimen C)

N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100

cALD HR-D (High-Risk, Regimen D)

유전성 대사 질환(IMD) - 반일치 유전성 질환 제외

치료법 설명을 참조하십시오.

줄기세포 이식

IMD 준비 요법

중증 골다공증(OP) - 반일치 유전성 골다공증 제외

치료법 설명을 참조하십시오.

골경화증 준비 요법만 해당

중증 골경화증(OP) 및 유전성 대사 장애(IMD)

-반일치 유전형에만 해당

치료법 설명을 참조하십시오.

골경화증 반일치형 준비 요법

개입 방법에 대한 설명을 참조하십시오.

cALD SR-A(표준 위험, 체제 A)

cALD SR-B(표준 위험, 요법 B)

cALD HR-D (고위험군, C 요법)

cALD HR-D (고위험군, D 요법)

Masonic Cancer Center, University of Minnesota

다발성 경화증 환자의 신체 활동을 위한 행동 중재(BIPAMS), 다발성 경화증 환자를 위한 웰니스(WellMS)

다발성 경화증 환자를 위한 좌식 신체 활동 중재 프로그램인 "SPIN" 프로그램 - 중재 조건, 다발성 경화증 환자의 장수를 위한 웰빙 프로그램(WELL) - 대조 조건

원격진료를 통한 다발성 경화증 임상 진료 제공, 기존 병원 방문을 통한 다발성 경화증 임상 진료 제공

가정 기반 자기 관리 및 인지 훈련은 다발성 경화증 환자의 삶을 변화시킵니다(HOBSCOTCH-MS)

오크렐리주맙을 복용한 다발성 경화증 환자는 인지 및 피로 평가를 받게 됩니다.

다발성 경화증 운동 지침(GEMS), FLEX 스트레칭 및 토닝 프로그램

다발성 경화증 환자의 신체 활동을 위한 행동 중재(BIPAMS)

MT2013-31: 대사 장애 및 중증 골경화증에 대한 동종 조혈모세포 이식