Spinal Muscular Atrophy

**Who might be able to join this trial:** - Your baby is a boy or girl who is between 1 day and 42 days old at the time of the first dose (the very first baby enrolled must be at least 7 days old) - Your baby was born between 37 and 42 weeks of pregnancy (if a single birth), or between 34 and 42 weeks (if a twin) - Yo...

**Who might be able to join this trial:** - The baby must be 6 weeks old or younger at the time of the first dose. - The baby must have a confirmed genetic test showing they have the type of SMA caused by a change or deletion on chromosome 5q. - The baby must have a genetic test confirming they have 2 or 3 copies of a...

**Who might be able to join this trial:** - A parent or guardian has given their signed permission for the child to take part, and the participant themselves has also agreed to join if appropriate for their age. - The participant has recently finished a related ("index") study — either by completing it normally or beca...

**Who might be able to join this trial:** - Your baby must be 6 weeks old or younger (no more than 42 days old) at the time they receive the study treatment - Your baby must be able to swallow thin liquids safely, as confirmed by a formal swallowing test - Your baby must have a nerve signal strength result (called a C...

**Who might be able to join this trial:** - You have been diagnosed with Spinal Muscular Atrophy (SMA) confirmed by a genetic test showing specific changes in the SMN1 gene, along with at least one copy of the SMN2 gene - You weigh between 8.5 kg (about 18.7 lbs) and 21 kg (about 46.3 lbs) at the time of the screening...

**Who might be able to join this trial:** - You have been diagnosed with Spinal Muscular Atrophy (SMA) - You are between 2 and 17 years old - You have already received at least four loading doses of nusinersen (Spinraza®), or have been taking risdiplam (Evrysdi®) for at least 3 months before the screening visit - You a...

Who might be able to join this trial: - You have been diagnosed with a rare disease, a disease where the number of people affected is unknown, or you have not yet received a diagnosis for your condition - You have been identified as a carrier of a rare or uncommon disease, meaning you carry the gene for a condition but...

Who might be able to join this trial: - You must have been diagnosed with a specific type of prostate cancer (adenocarcinoma) confirmed by a tissue or cell sample, without certain aggressive features (small cell or neuroendocrine). - You must have prostate cancer that has spread to other parts of the body, shown by bon...

**Who might be able to join this trial:** - You are a man aged 18 or older who has been diagnosed with prostate cancer that has spread to other parts of the body (such as bones or soft tissue), either newly diagnosed or returning after previous treatment, and your cancer does not have certain rare, aggressive features...

**Who might be able to join this trial:** - You must be 18 years old or older (or the legal adult age in your country) at the time of signing the consent form. - You must have been diagnosed with prostate cancer (a specific type called adenocarcinoma), confirmed by a tissue or cell sample. - Your prostate cancer must...

**Who might be able to join this trial:** - The baby was born after a full-term pregnancy (between 37 and 42 weeks) - The baby has been officially diagnosed with Spinal Muscular Atrophy (SMA) - The baby has exactly 2 copies of a specific gene called SMN2 - The baby's weight is at or above the 3rd percentile (a minimum...

Who might be able to join this trial: - People who have been diagnosed with certain neurodegenerative disorders (diseases that affect the nervous system) and are either scheduled to have surgery for medical reasons or are willing to donate one or a few hairs removed whole from the scalp or another area of the body. - P...

Who might be able to join this trial: - You have been diagnosed with a spinal cord injury, a brainstem stroke, muscular dystrophy, ALS (Lou Gehrig's disease), or a similar condition affecting movement - You have partial or complete loss of movement and feeling in both your arms and legs (confirm with trial site) - You...

Who might be able to join this trial: - The baby must be a newborn born in Taiwan who is already receiving the standard newborn screening tests recommended by Taiwan's Ministry of Health and Welfare. - The baby's parents or legal guardian must agree to have the baby tested for SMA (Spinal Muscular Atrophy) as part of t...

Who might be able to join this trial: - You have been diagnosed with ALS (Lou Gehrig's disease) or a related condition, such as primary lateral sclerosis or progressive muscular atrophy. - You receive your medical care at a clinic or hospital that uses a health records system called Epic. - You are able and willing to...

**Who might be able to join this trial:** - You are older than 21 years of age - You have limited ability to use your arms or hands due to a stroke, ALS (Lou Gehrig's disease), multiple sclerosis, a neck/spinal cord injury, muscular dystrophy, muscle disease, or severe nerve damage - Your disability is severe enough th...

**Who might be able to join this trial:** - You have been diagnosed with axial spondyloarthritis (a type of inflammatory spine condition) according to specific medical classification guidelines (confirm with trial site) - You have never previously been treated with a "targeted therapy" (a specific type of modern medic...

Who might be able to join this trial: - People diagnosed with Spinal Muscular Atrophy (SMA) type I, II, or III - People who carry the gene change linked to SMA but do not have symptoms themselves - Family members of someone who has SMA or carries the SMA gene - Healthy volunteers who are not related to anyone with SMA...

Who might be able to join this trial: - You have been treated with a gene therapy called OAV-101 (also known as Zolgensma) and have been diagnosed with Spinal Muscular Atrophy (SMA) confirmed by genetic testing, no matter when you were diagnosed. - You (or your parent/guardian, if applicable) have agreed and given perm...

**Who might be able to join this trial:** - You have been diagnosed with Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene (types 1 to 4), confirmed by a genetic test. - You have been treated at a specialist SMA centre in France between September 1, 2016 and August 31, 2024. - For the part of the study lo...

Who might be able to join this trial: - Anyone who has been diagnosed with SMA (Spinal Muscular Atrophy), or is currently waiting for a diagnosis to be confirmed, may be eligible. - Diagnosis of SMA needs to be confirmed through genetic testing results (or may still be pending at the time of joining). Who may not be a...

**Who might be able to join this trial:** - You are between 8 and 99 years old and have been diagnosed with androgen insensitivity syndrome (AIS), either through a genetic test showing a change in the androgen receptor gene, or through a clinical diagnosis based on specific physical and biological signs - You identify...

**Who might be able to join this trial:** **If you have SBMA (Spinal Bulbar Muscular Atrophy):** - You are willing to follow all study procedures and are available for the full duration of the study - You are male and over 18 years old - Your SBMA diagnosis has been confirmed through genetic testing - You are able to...

**Who might be able to join this trial:** **If you are a patient:** - You have been diagnosed with ALS (in any confirmed stage or type), ALS with frontotemporal dementia (ALS-FTD), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), or you carry a known ALS gene mutation even if you have no symptoms y...

**Who might be able to join this trial:** - You regularly and consistently wear a leg brace (on one or both legs) that supports your knee and/or ankle, due to a neurological condition, muscle disease, bone/joint condition, or an injury - You are able to understand and follow the study instructions, and are willing to g...

**Who might be able to join this trial:** *For healthy volunteers:* - Be a similar age and gender to the patient groups in the study - Be physically able to take part in the experiment *For patients:* - Have a diagnosis of ALS, PLS, PMA, SMA, Polio, or MS - Be able to understand and agree to take part in the study on...

**Who might be able to join this trial:** - You are between 18 and 80 years old. - You have been diagnosed with ALS (a condition affecting the nerve cells that control muscles), confirmed by a specialist in neurological diseases. - You have ALS and have completely lost the ability to speak, or have severely reduced spe...

**Who might be able to join this trial:** - You are willing to sign a consent form and agree to follow all study procedures for the full duration of the trial. - You are between 3 and 17 years old. - You have a walking difficulty related to the knee joint, caused by cerebral palsy, muscular dystrophy, spina bifida, or...

**Who might be able to join this trial:** - The child's parent or legal guardian must be willing to sign a consent form to allow participation in the study - The child must be younger than 240 days old (about 8 months) at the time the consent form is signed - The child must have a confirmed genetic diagnosis of spinal...

Who might be able to join this trial: - All patients may be considered for this trial. Who may not be able to join: - People who are currently enrolled in other clinical trials may not be able to join. Important: Always verify eligibility with the trial site directly before applying.

Who might be able to join this trial: - People who have mutations (changes) in a gene called SMN1 Who may not be able to join: - People who are unable to give their consent to take part in the trial Important: Always verify eligibility with the trial site directly before applying.

**Who might be able to join this trial:** - You are currently pregnant, or were pregnant during the relevant time period, and are already registered in one of these specific patient registries in the US, UK, Germany, Austria, or Switzerland - You have genetic test results confirming a diagnosis of spinal muscular atrop...

**Who might be able to join this trial:** - The baby must be 6 months old or younger on the day they receive the treatment - The baby must have been diagnosed with Type 1 Spinal Muscular Atrophy (SMA) confirmed by a genetic test showing changes in both copies of the SMN1 gene and exactly 2 copies of the SMN2 gene - Sy...

**Who might be able to join this trial:** - You must be under 2 years old at the time of joining the trial - Your child must have a confirmed diagnosis of spinal muscular atrophy (SMA) caused by a specific genetic change affecting the SMN1 gene - Your child must have exactly two copies of the SMN2 gene, confirmed by a...

**Who might be able to join this trial:** - You must be under 2 years old at the time of joining the study - Your child must have a confirmed diagnosis of spinal muscular atrophy (SMA) caused by a specific genetic change affecting the SMN1 gene - Your child must have exactly two copies of a related gene called SMN2, co...

**Who might be able to join this trial:** - You are 3 years old or older - You have been diagnosed with Spinal Muscular Atrophy (SMA) and are a candidate for a drug called Spinraza (nusinersen) - You have difficulty receiving spinal injections (lumbar punctures) safely — either because breathing problems or other heal...

**Who might be able to join this trial:** - You have been diagnosed with ALS (also called Lou Gehrig's disease), at any level of certainty — suspected, possible, probable, or definite - You have been diagnosed with another motor neuron disorder, such as Kennedy's disease, Spinal Muscular Atrophy (SMA), Primary Lateral...

**Who might be able to join this trial:** - A parent or legal guardian must have signed the electronic informed consent form on the child's behalf. - The child must have been diagnosed with Spinal Muscular Atrophy (SMA), confirmed by a genetic test showing a specific gene mutation, and must have SMA Type 1 or a certai...

**Who might be able to join this trial:** - You are between 18 and 80 years old - You have been diagnosed with a spinal cord injury, a brainstem stroke, muscular dystrophy, ALS (Lou Gehrig's disease), or another condition affecting movement and motor control - You have significant weakness or paralysis affecting both y...

**Who might be able to join this trial:** - You are pregnant and at least 9 weeks along (based on your last period) - You are carrying one baby (not twins or more) - You are having an invasive prenatal test (such as amniocentesis) because your family has a history of a specific inherited genetic disorder affecting one...

**Who might be able to join this trial:** - The child must be an infant with muscle weakness (sometimes called a "floppy baby") who has been officially diagnosed with Spinal Muscular Atrophy (SMA) through a genetic test showing a change or missing section in the SMN1 gene. - The child must be receiving standard medical...

**Who might be able to join this trial:** - You are male and have been diagnosed with SBMA (Spinal and Bulbar Muscular Atrophy) confirmed by a genetic test showing a specific gene repeat number of 38 or higher - You are between 18 and 75 years old - You are experiencing one or more of the following: muscle wasting, wea...

Who might be able to join this trial: - You must be willing and able to sign a consent form agreeing to take part in the study - You must be male and between 19 and 49 years old with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) - You must not currently be taking any medication or treatment specifically design...

**Who might be able to join this trial:** - You have been diagnosed with Duchenne Muscular Dystrophy (DMD), confirmed by a genetic test report - You are at least 6 years old - You are able to walk - You have been on a stable steroid (glucocorticoid) treatment for more than 3 months **Who may not be able to join:** - Y...

**Who might be able to join this trial:** - You have been diagnosed with infantile or juvenile spinal muscular atrophy (SMA), confirmed by genetic testing - You are currently being treated with a medication that helps the body produce SMN protein (such as nusinersen, risdiplam, or onasemnogene abeparvovec) - You are be...

**Who might be able to join this trial:** - You (or your child) must agree to take part by signing a consent form — if the participant is a child, a parent or legal guardian can sign on their behalf - You (or your child) must have a confirmed diagnosis of Spinal Muscular Atrophy (SMA, type 1–4), proven by a genetic tes...

**Who might be able to join this trial:** - You have been formally diagnosed with a neuromuscular disease (such as muscular dystrophy, spinal muscular atrophy, ALS, Charcot-Marie-Tooth disease, or a similar condition affecting the muscles or nerves), confirmed through genetic testing, a muscle biopsy, or nerve/muscle t...

**Who might be able to join this trial:** - People who have signed and dated a written consent form before any study procedures begin - Women aged 18 years or older - People who have a biopsy-confirmed diagnosis of breast cancer that has come back, cannot be surgically removed, or has spread — and whose cancer tests p...

**Who might be able to join this trial:** - You have been diagnosed with spinal muscular atrophy (SMA) caused by a problem on chromosome 5 (called 5q SMA), confirmed by a genetic test - You are between 8 and 18 years old at the time of screening - You are able to sit up on your own without any support for at least 10 s...

**Who might be able to join this trial:** - You must be 2 years of age or older at the time of signing the consent form - You must have a confirmed genetic and clinical diagnosis of Type 3 Spinal Muscular Atrophy (SMA), caused by a specific gene change on chromosome 5 - Your score on a standard motor skills test (calle...

**Who might be able to join this trial:** - You have been confirmed through genetic testing to have SMA (Spinal Muscular Atrophy), with a specific change or deletion in the SMN1 gene - You have two, three, or four copies of the SMN2 gene - You are between 5 and 20 years old - If you cannot walk independently: you can...

Who might be able to join this trial: - You have a rare disease (one that affects fewer than 200,000 people in the United States), whether it has been confirmed by a doctor or is only suspected. Examples include ALS, cystic fibrosis, sickle cell disease, Huntington's disease, Duchenne muscular dystrophy, early-onset Al...

Who might be able to join this trial: - You are already taking nusinersen (a medication for SMA) at a dose of 12 mg every 4 months as a regular ongoing treatment. - You must already be enrolled in a related study called the PIERRE study in order to take part in this trial. Who may not be able to join: - You are curren...

Who might be able to join this trial: - You have been diagnosed with spinal muscular atrophy (SMA) confirmed by a genetic test - You are 6 years old or older - You have not had any bone or joint surgery in the 6 months before joining the trial - You (or your parent or legal guardian) are willing and able to sign a cons...

**Who might be able to join this trial:** **For people with a spine condition:** - You have been diagnosed with Ankylosing Spondylitis or a related spine condition called non-radiographic Spondyloarthritis, confirmed using standard medical criteria - You are between 18 and 65 years old - You are willing to give your c...

Who might be able to join this trial: - You are an adult who can walk (even with the help of a walking aid or support) and have been diagnosed through genetic testing with a muscular dystrophy or myopathy (such as a condition affecting the muscles from birth or developing later in life), spinal muscular atrophy (SMA),...

**Who might be able to join this trial:** - You are either 65 years of age or older (for the older adult group) or between 18 and 39 years old (for the younger adult group) - You are able to walk on your own without using a cane, walker, or any other assistive device - You have enough muscle strength in your main muscl...

**Who might be able to join this trial:** - You (or your child) have been diagnosed with Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA), **or** you are a healthy person with no neuromuscular disease (to take part as a comparison participant) - You are older than 5 years of age - You are able to breat...

**Who might be able to join this trial:** - You have a confirmed genetic diagnosis of Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA), or you are a healthy volunteer (used as a comparison group) - You are able to walk at least 25 meters (about 82 feet) on your own, without help - If you have DMD: you a...

**Who might be able to join this trial:** - You are a male with a confirmed diagnosis of SBMA (Spinal and Bulbar Muscular Atrophy), confirmed by genetic testing showing a specific gene repeat count of 38 or more - You have muscle weakness in your arms or legs, muscle wasting, or weakness in the muscles used for swallo...

**Who might be able to join this trial:** - You have been diagnosed with spinal muscular atrophy (SMA) caused by a problem with the SMN1 gene (either a missing copy or a mutation), regardless of how many copies of the SMN2 gene you have - You are currently taking nusinersen (Spinraza) at a dose of either 12 mg or 28 mg...

**Who might be able to join this trial:** - You have been diagnosed with Type 2 Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene, confirmed by genetic testing and symptoms. - You have been regularly receiving the medication nusinersen for more than one year before the screening visit. - You have not take...

**Who might be able to join this trial:** - You have been diagnosed with Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene, confirmed by a DNA/genetic test. - You are able to give your consent to take part in the study. - You have at least a basic level of hand and arm function — for example, you can hold...

**Who might be able to join this trial:** - You are 16 years old or older (as this is when the transition to adult care begins). - You have been diagnosed with a specific type of spinal muscular atrophy called 5q SMA, confirmed by a genetic test. - You (or your legal guardian, if applicable) have signed a consent form...

**Who might be able to join this trial:** - You are willing to sign a consent form and agree to follow all study procedures for the full duration of the trial. - You are between 5 and 25 years old. - You either have a walking difficulty caused by cerebral palsy (CP), muscular dystrophy (MD), spina bifida (SB), or an i...

**Who might be able to join this trial:** - You must be under 2 years old at the time of signing up for the trial - Your baby must have been born at 35 weeks of pregnancy or later, and weighed at least 2.0 kg (about 4.4 lbs) at birth - Your child must have a confirmed diagnosis of Spinal Muscular Atrophy (SMA) caused...

**Who might be able to join this trial:** - **Children showing symptoms:** Your child has been diagnosed with SMA Type 1 through genetic testing, showing changes in the SMN1 gene and having up to 3 copies of the SMN2 gene. - **Children showing symptoms:** Your child is between 2 weeks and 12 months old at the time of...

Who might be able to join this trial: - You must be able to walk at least 10 meters (about 33 feet) on your own, without help from another person or a mobility aid. - You must have a confirmed diagnosis of one of the conditions being studied, based on appropriate testing such as genetic tests or clinical assessment. -...

**Who might be able to join this trial:** - The baby must be 42 days old or younger when they receive their first dose of the study drug. - The baby must have a genetic test confirming they have Spinal Muscular Atrophy (SMA) caused by a specific change or deletion on chromosome 5. - The baby must have either 2 or 3 co...

Who might be able to join this trial: - You have been diagnosed with SMA (Spinal Muscular Atrophy) and are 18 years old or older Who may not be able to join: - You are deceased Important: Always verify eligibility with the trial site directly before applying.

Who might be able to join this trial: - You have been diagnosed with a type of Spinal Muscular Atrophy (SMA) that has been confirmed through genetic testing to be caused by a change on chromosome 5q. - You (or your caregiver) have given written agreement to take part in the trial (note: this requirement may differ for...

Who might be able to join this trial: - You must be willing and able to sign a written consent form to take part in the study. - You must have a genetic test confirming you have Spinal Muscular Atrophy (SMA), shown by a specific change or missing section in the SMN1 gene. - Your doctor must believe that the treatment b...

**Who might be able to join this trial:** - Your child has been confirmed through genetic testing to have SMA (Spinal Muscular Atrophy), their SMN2 copy number is known, and they were identified through newborn screening, family screening, or found to have the condition before showing any symptoms — and they are eithe...

**Who might be able to join this trial:** - You are between 5 and 17 years old (inclusive), a parent or guardian can sign the consent form on your behalf, and you are able to follow the study requirements - You have been officially diagnosed with 5q Spinal Muscular Atrophy (SMA) confirmed through genetic testing - You...

**Who might be able to join this trial:** - You are between 18 and 65 years old - You are able to walk around (not confined to a wheelchair or bed) - You are willing to give your written agreement to take part in the study - You are covered by a social security or health insurance system - *(For people with a neuromusc...

Who might be able to join this trial: - You must be an adult (18 years or older) - You must have either 1 or 3 copies of a gene called SMN1 (this would place you in the control group), with any number of copies of another gene called SMN2, OR you must have 2 copies of the SMN1 gene arranged in a specific way on one chr...

Who might be able to join this trial: - You have been diagnosed with a specific type of spinal muscular atrophy called 5q SMA - You are between 8 and 50 years old - You are able to walk 10 meters (about 33 feet) on your own without help from another person or a walking aid - You may join whether or not you are currentl...

**Who might be able to join this trial:** - You are between 15 and 60 years old (inclusive) at the time you give your consent to participate - You have a confirmed genetic diagnosis of 5q Spinal Muscular Atrophy (SMA), shown through genetic testing - You have physical signs and symptoms that match a diagnosis of SMA -...

Who might be able to join this trial: - You have had, or currently have, a specially trained personal care assistant who is certified to help with breathing support, at some point between 2016 and 2025. Who may not be able to join: - You do not have a tracheostomy (a surgically made opening in the throat used for brea...

**Who might be able to join this trial:** - You are 18 years of age or older - You have been diagnosed with at least one of the following conditions: ALS (amyotrophic lateral sclerosis), primary lateral sclerosis, progressive muscle atrophy, spinal muscular atrophy, post-polio syndrome, inclusion body myositis, Pompe...

**Who might be able to join this trial:** - You have been officially diagnosed with SMA (Spinal Muscular Atrophy) - You are between 5 and 17 years old - You are able to attend and complete all required check-ins and assessments before and after the study - You can understand and speak English - You are able to follow i...

**Who might be able to join this trial:** - You have significant weakness or loss of movement in both arms (and possibly legs), or severe problems with coordination, due to a confirmed medical condition. - Your condition is caused by a brainstem stroke, a traumatic spinal cord injury, or a progressive muscle or nerve...

**Who might be able to join this trial:** *For children with SMA (Test group):* - Your child has been confirmed through genetic testing to have SMA, and the number of their SMN2 gene copies is known - Your child was identified through newborn screening and is receiving treatment for SMA, OR was diagnosed because a sib...

**Who might be able to join this trial:** **Cohort 1 – Survey study (people with SMA and caregivers):** - You have been diagnosed with SMA that has been confirmed by genetic testing, and you are between 12 and 18 years old - You have a good understanding of English, or someone who can help you understand English well...

Who might be able to join this trial: - People who are between 18 and 65 years old - People who have been admitted to an intensive care unit because of a brain-related condition, such as bleeding in or around the brain, a brain tumour, or a stroke - People (or their legal representative) who have given formal written a...

**Who might be able to join this trial:** - People who have been diagnosed with prostate cancer (a specific type called adenocarcinoma, without small cell features), confirmed through a tissue or cell sample - People whose cancer has continued to grow or spread despite having their testosterone lowered through surgery...