Biogen, Inc.

In this study, researchers will learn more about the study drugs diroximel fumarate (DRF) and dimethyl fumarate (DMF) in children with MS who may be experiencing relapses.
Participants will be divided into 2 groups based on their weight:
* Group A will include children who weigh 40 kilograms (kg) or less. They will receive DRF in both Part 1 and Part 2 of the study.
* Group B will include children who weigh more than 40 kg. They will be randomly assigned to receive DRF, DMF, or fingolimod. Fingolimod is a drug already used to treat MS in adults. In Part 2, they will receive DRF.
This is a 2-part study. Part 1 treatment will last 96 weeks. Participants who complete Part 1 may move to Part 2. Part 2 is an extension period. Treatment will last another 96 weeks and will help researchers learn about the long-term safety and effects of treatment.
The main goal of the study is to learn about the safety of DRF and DMF and compare their effect on relapses and brain lesions with fingolimod.
The main questions researchers want to answer are:
* How many participants have adverse events and serious adverse events?
* How often do participants relapse after treatment with DRF and DMF compared to fingolimod?
Researchers will take brain imaging scans to check for any new areas of brain inflammation and compare the brain lesions before and after treatment.
Researchers will also measure the amount of drug in the blood to understand how the body processes it. To check safety, they will monitor participants' growth and development, and compare changes in heart tests, vital signs, and lab tests. They will also use rating scales to monitor depression symptoms.
The study will be done as follows:
Part 1 (Treatment Period)
* After screening, participants will join Part 1 and be divided into 2 groups based on their weight.
* Participants in Group A will receive DRF.
* Participants in Group B will be randomly assigned to receive either DRF, DMF, or fingolimod.
* Neither the researchers nor the participants will know which drug or dose the participants will receive in Group B. Participants in Group B will also receive a placebo so they do not know which drug is being given. A placebo looks like a study drug but contains no real medicine.
* All study drugs will be taken by mouth, once or twice a day. Participants who take DRF or DMF will start with a lower dose during the 1st week, then move to a standard dose.
* Treatment in Part 1 will last for 96 weeks. Participants will have up to 11 study visits and 7 phone calls.
* Participants who do not move onto Part 2 will also have a safety follow-up period of 4 to 8 weeks. This will include 1 study visit and 1 phone call.
* The total length of Part 1, including the screening, treatment, and follow-up, will be up to 108 weeks.
Part 2 (Extension Period)
* Participants who complete Part 1 can move on to Part 2 of the study.
* All participants in Part 2 will receive DRF by mouth for 96 weeks.
* Participants will have up to 10 more study visits and 1 telephone call during treatment.
* They will also have a 4-week safety follow-up, including 1 study visit and 1 phone call.
* The total length of Part 2 will be up to 100 weeks.

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will have taken BIIB141 at any time during pregnancy and/or while breastfeeding or pumping up through the first year after delivery. Participants can join this study on their own or they may be enrolled by their regular doctors. This study is also known as the "SKYCLARYS (Omaveloxolone) Pregnancy and Lactation Surveillance Program."
The main objective of this study is to learn more about how BIIB141 may affect pregnancy, as well as any effects on the health of the mother and of the baby during its first year of life.
The main question researchers want to answer in this study is:
· Does taking BIIB141 during pregnancy or breastfeeding lead to any major birth defects?
Researchers will also learn more about:
* Does taking BIIB141 during pregnancy or breastfeeding lead to any minor birth defects?
* Does taking BIIB141 during pregnancy or breastfeeding affect the following:
* Gestational diabetes, a disease that can happen during pregnancy that affects how your body uses sugar
* Pre-eclampsia, a pregnancy-related high blood pressure disease
* Unborn baby being small for its expected age (usually in weeks)
* Loss of an unborn baby
* Live birth
* Premature birth
* Loss of a newborn
* Growth or developmental delays in the baby
* Serious illness in the baby resulting in hospitalization
* Serious infections in the baby, or ones in babies with a weakened immune system
This study will be done as follows:
* Participants will join the study after signing an informed consent form, also known as an ICF.
* During the study, health information from the participants' regular visits to their doctor will be collected based on whether participant joined the study while pregnant or after the baby is born.
* Each participant will be in the study for up to 1 year after the birth of their child, unless they decide to leave early. Overall, this study is expected to last at least 10 years.

In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with onasemnogene abeparvovec (OA) after being diagnosed with SMA.
Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein.
OA works by replacing the missing or abnormal SMN1 gene. Sometimes, OA treatment may not work as well as expected. As a result, researchers are exploring whether giving another drug after OA could lead to better outcomes for people with SMA.
In this study, participants will have 2 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. They will also have received treatment with OA by the time they were 42 days old and before showing any symptoms of SMA.
The main goal of the study is to learn more about the safety of giving salanersen to babies after OA treatment. Researchers will also learn more about whether salanersen can help make SMA symptoms less serious.
The main question researchers want to answer in this study is:
• How many participants have adverse events and serious adverse events after treatment?
Researchers will also learn more about:
* The effects on participants' motor symptoms and how many new movement milestones participants achieve.
* How many participants stay free of SMA symptoms.
* How much neurofilament protein is found in the blood after treatment.
* How much salanersen gets into the fluid surrounding the brain and spinal cord.
* How much salanersen gets into the blood. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones and Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestones.
The study will be done in 2 parts. Part A will last 1 year while Part B will last up to 4 years.
The study will be done as follows:
* First, participants will be screened to check if they can join the study. The screening period will be up to 6 months. Participants must have received OA treatment before the age of 42 days and started screening within 6 months of the OA dose.
* Participants will be assigned to 1 of 2 treatment groups by chance. This is a "double blind" study which means neither the participants, study doctor, nor site staff will know which treatment group the participants are assigned to.
* In this study, salanersen will be given as an intrathecal injection, which is an injection into the fluid surrounding the spine. This is done by a procedure called a lumbar puncture (LP) which involves inserting a needle into the lower back into the space around the spinal cord.
* During Part A, one group will receive 80 milligrams (mg) of salanersen while another group receives a sham (fake) procedure. This means that a small needle prick will be done, but no injection will be given.
* For each participant, the first visit of Part A will be 6 months after they receive OA treatment.
* Part A will have up to 6 clinic visits and 2 phone calls and last up to 1 year.
* During Part B, both groups of participants will receive 80 mg of salanersen once a year.
* Part B will have up to 12 clinic visits and 14 phone calls and last up to 4 years.
* In total, participants will be in the study for up to 5 and a half years.