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26 Spinal Muscular Atrophy Trials near Miami, FL
Power is an online platform that helps thousands of Spinal Muscular Atrophy patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerThis study will evaluate if Ursolic Acid supplementation may be effective in reducing muscle loss and improving blood sugar control in the SCI community.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Breastfeeding
20 Participants Needed
Taldefgrobep Alfa for Spinal Muscular Atrophy
Miami, FloridaThis trial will test if taldefgrobep alfa can help people with SMA who are already on other treatments. The drug works by blocking a protein that stops muscle growth, potentially making muscles stronger and improving movement. Taldefgrobep alfa has shown promise in increasing muscle mass and function in SMA patients.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Age:4 - 21
Sex:All
269 Participants Needed
Long-Term Risdiplam for Spinal Muscular Atrophy
Orlando, FloridaA multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
An optional sub study will assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. Approximately 39 participants from the main study are planned to be enrolled in the sub study.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:2+
Sex:All
402 Participants Needed
Nusinersen for SMA
Orlando, FloridaIn this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on children under the age of 3 who were previously treated with the gene therapy onasemnogene abeparvovec but are still facing health challenges related to their disease.
The main goal of the study is to learn about the effect nusinersen has on muscle and movement ability (motor function). The main question researchers want to answer is:
- What score do participants have on the HINE Section 2 Motor Milestones test after treatment?
The Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones is an assessment that tests movements in different positions. This includes grasping, kicking, head control, rolling, sitting, crawling, standing, and walking.
Researchers will use a group of tests to study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing.
Researchers will also learn more about the safety of nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests.
The study will be done as follows:
* Participants will be screened to check if they can join the study.
* Each participant will receive 4 initial doses of 12 mg of nusinersen on Days 1, 15, 29, and 64 of the Treatment Period. Then, they will receive 12 mg doses once every 4 months.
* The total number of doses of nusinersen will be 9.
* Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back.
* The treatment period will last for up to 95 weeks (close to 2 years).
* There will be a follow-up safety period that lasts about 4 months.
* In total, participants will have up to 14 study visits. Participants will stay in the study for up to 115 weeks.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:2 - 36
Sex:All
46 Participants Needed
Nusinersen for Spinal Muscular Atrophy
Orlando, FloridaThe primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:6 - 6
Sex:All
25 Participants Needed
Long-Term Apitegromab for Spinal Muscular Atrophy
Orlando, FloridaThis trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+
Sex:All
238 Participants Needed
RO7204239 + Risdiplam for Spinal Muscular Atrophy
Orlando, FloridaThis trial tests the safety and effectiveness of combining two treatments, RO7204239 and risdiplam, for patients with spinal muscular atrophy (SMA). Risdiplam helps the body produce a protein needed for muscle movement, while RO7204239 allows muscles to grow bigger and stronger. The study includes SMA patients to see if this combination improves their muscle function and overall health.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:2 - 25
Sex:All
259 Participants Needed
Risdiplam for Presymptomatic Spinal Muscular Atrophy
Orlando, FloridaThis trial is testing a medicine called risdiplam, which is taken by mouth. It aims to help infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but are not yet showing symptoms. The medicine works by helping the body make more of a protein that muscles need to stay healthy. The goal is to see if early treatment can prevent or lessen the severity of SMA.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 6
Sex:All
26 Participants Needed
Risdiplam for Spinal Muscular Atrophy
Orlando, FloridaThis is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:6 - 60
Sex:All
174 Participants Needed
NMD670 for Spinal Muscular Atrophy
Gainesville, FloridaThe purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18 - 75
Sex:All
Key Eligibility Criteria
Disqualifiers:Scoliosis, Neuromuscular Diseases, Others
54 Participants Needed
Risdiplam for Spinal Muscular Atrophy
Gainesville, FloridaThis is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:3 - 24
Sex:All
Key Eligibility Criteria
Disqualifiers:Invasive Ventilation, Feeding Tube, Others
Must Not Be Taking:Antisense Oligonucleotides, Splicing Modifiers
28 Participants Needed
Risdiplam for Spinal Muscular Atrophy
Gainesville, FloridaThis is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:3 - 24
Sex:All
Key Eligibility Criteria
Disqualifiers:Invasive Ventilation, Tracheostomy, Feeding Tube, Others
Must Not Be Taking:Antisense Oligonucleotides, Splicing Modifiers
28 Participants Needed
High-Dose Nusinersen for Spinal Muscular Atrophy
Atlanta, GeorgiaIn this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on teenagers and adults who are unable to walk on their own and who have previously taken another drug for SMA called risdiplam.
The main goal of this study is to learn about the effect of high dose (HD) nusinersen on muscle and movement ability (motor function) in SMA. The main question that researchers want to answer is:
- How do the scores of a movement test called the Revised Upper Limb Module change from the start of treatment?
The Revised Upper Limb Module is a test used to measure a participant's ability to do specific tasks that involve their shoulders, arms, wrist, elbows, and hands. It measures the changes in their abilities over time.
Researchers will also learn more about the safety of HD nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests.
The study will be done as follows:
* Participants will be screened to check if they can join the study.
* After screening, participants will enter the Core Treatment period.
* At the start of the Core Treatment period, they will receive 2 "loading" doses of nusinersen. These are 50 mg doses of nusinersen given 2 weeks apart.
* Afterwards, they will continue to receive "maintenance" doses of nusinersen once every 4 months. These doses will be 28 mg.
* The Core Treatment period will last about 2 years, with a follow-up visit 4 months after the last dose.
* Participants who complete the Core Treatment period will have the option to continue receiving 28 mg of nusinersen in the Long-Term Extension (LTE) period for about 2 years. There will also be a follow-up visit 4 months after the last dose.
* Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back.
* In total, participants will have up to 18 study visits. They will also be called by researchers after each dose of nusinersen.
* Participants will stay in the study for about 4.5 years if they complete both the Core Treatment and LTE periods.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:15 - 50
Sex:All
Key Eligibility Criteria
Disqualifiers:Major Illness, Active Infection, Others
Must Be Taking:Risdiplam
45 Participants Needed
Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
Durham, North CarolinaThis is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
85 Participants Needed
OAV101 for Spinal Muscular Atrophy
Norfolk, VirginiaThis is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:0 - 100
Sex:All
175 Participants Needed
OAV101 for Spinal Muscular Atrophy
Norfolk, VirginiaTo evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to \< 18 years of age over a 15 month trial duration.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2 - 17
Sex:All
127 Participants Needed
Nusinersen for Spinal Muscular Atrophy
Memphis, TennesseeIn this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203.
The main goal of the study is to learn about the long-term safety of nusinersen. The main questions researchers want to answer are:
* How many participants have adverse events and serious adverse events during the study?
* How do the results of electrocardiograms (ECGs), vital signs, and laboratory tests including blood and urine tests change after treatment?
* How many participants have a low platelet count after treatment?
* How many participants had a change in the time it took for their heart to recharge between beats after treatment?
* How does each participant's height and other measures of growth change after treatment?
* How much do the results of neurological exams that check movement, reflexes, and brain function change after treatment?
Researchers will also learn about the effect of nusinersen on mobility using various tests. They will study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing.
The 232SM302 study will be done as follows:
* Participants will be screened to check if they can join the study.
* Participants will receive their 1st dose of nusinersen in this study about 4 months after their final dose in the parent study.
* Each participant will receive nusinersen once every 4 months during the treatment period.
* Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back.
* The treatment period will last for up to 64 months (1921 days).
* There will be a follow-up safety period that lasts from 4 to 8 weeks.
* In total, participants will have up to 19 study visits. Participants will stay in the study for close to 6 years.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+
Sex:All
115 Participants Needed
Nusinersen for Spinal Muscular Atrophy
Houston, TexasIn this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study.
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:
* What is the highest amount of nusinersen found in the blood after dosing?
* How much nusinersen is found in the blood over the first 24 hours after dosing?
The PIERRE-PK study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study.
* Participants will receive a dose of nusinersen by lumbar puncture.
* The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study.
* Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study.
* Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose.
* The total study duration for each participant in the PIERRE-PK study will be up to 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:3+
Sex:All
Key Eligibility Criteria
Disqualifiers:Other Trials, Nusinersen Naive, Pregnant, Others
Must Be Taking:Nusinersen
55 Participants Needed
Gene Therapy for Spinal Muscular Atrophy
Columbus, OhioThis trial uses a harmless virus to deliver a healthy gene directly into the spinal fluid of patients with diseases caused by IGHMBP2 gene defects. The virus helps correct the genetic issues by bringing the healthy gene to the affected cells. This approach has been shown to improve conditions in similar diseases in animals.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 1, 2
Age:2 - 14
Sex:All
10 Participants Needed
Risdiplam for Spinal Muscular Atrophy
Strasburg, PennsylvaniaRisdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment. The schedule of assessments (SOAs) carry over seamlessly for the cohort from studies done while treated with nusinersen and continue to track the most informative outcomes from that trial (e.g. nine hole peg test and grip strength), while adding the Box and Block Test (BBT) as an additional measure of upper limb endurance and function.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:2 - 35
Sex:All
10 Participants Needed
Neurosleeve for Arm Weakness/Paralysis
Wilmington, DelawareThis trial is testing the NuroSleeve, a wearable device that helps people with weak or paralyzed arms move them using electrical signals. It is aimed at those who haven't fully recovered with standard therapies. The device detects small muscle movements and uses them to activate the brace and stimulate muscles, helping with arm movement. Electrical stimulation has been explored in various contexts, including improving arm and hand function in individuals with spinal cord injuries and post-stroke patients.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:4+
Sex:All
Key Eligibility Criteria
Disqualifiers:Visual Impairment, Orthopedic Conditions, Others
Must Not Be Taking:Bupropion
20 Participants Needed
Risdiplam for Spinal Muscular Atrophy
Gordonville, PennsylvaniaThis study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 19
Sex:All
Key Eligibility Criteria
Disqualifiers:SMA Type 0, Blood Pressure Abnormalities, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers, MATE Substrates
10 Participants Needed
Spinal Cord Stimulation for Spinal Muscular Atrophy
Pittsburgh, PennsylvaniaSpinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with Type 2, 3, or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 2, 3, or 4 SMA aged 16 or older that show quantifiable motor deficits of the upper body. The investigators will then implant the subjects with percutaneous, linear spinal leads near the cervical spinal cord for a period of up to 29 days. Although these leads are not optimized for motor function but rather for their clinically approved indication of treating pain, the investigators believe they provide a safe technology enabling our team to perform scientific measurement necessary to evaluate potential for effects of SCS in motor paralysis with SMA. After the end of the study, the leads will be explanted.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:16 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Spinal Deformation, Joint Contractures, Others
Must Be Taking:SMN Inducing Therapies
20 Participants Needed
Spinal Cord Stimulation for Spinal Muscular Atrophy
Pittsburgh, PennsylvaniaThis trial tests if spinal cord stimulation (SCS) can improve leg movement in people with Type 3 or 4 Spinal Muscular Atrophy (SMA) who can stand but have motor deficits. SCS uses electrical pulses to help the spinal cord communicate better with the brain, potentially improving muscle control and strength. Spinal cord stimulation (SCS) has been shown to improve motor performance in various conditions, including certain types of muscle weakness.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:16 - 64
Sex:All
3 Participants Needed
Exergaming for Spinal Muscular Atrophy
Toronto, OntarioThis is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational satisfaction of children and youth (8-18 years old age) with spinal muscular atrophy (SMA).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:8 - 18
Sex:All
Key Eligibility Criteria
Disqualifiers:Severe Scoliosis, Contractures, Others
Must Be Taking:Disease-modifying Therapy
22 Participants Needed
In this study, researchers will learn about a study drug called BIIB115 in healthy male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
The main objective of the study is to learn more about the safety of BIIB115 and how participants respond to different doses of BIIB115. The main question researchers want to answer is:
How many participants have adverse events and serious adverse events during the study? Adverse events are unwanted health problems that may or may not be caused by the study drug.
Researchers will also learn more about how the body processes BIIB115. They will do this by measuring the levels of BIIB115 in both the blood and the cerebrospinal fluid, also known as the CSF. This is the fluid around the brain and spinal cord.
The study will be split into 2 parts - Part A and Part B.
During Part A:
* After screening, healthy volunteers will be randomly placed into 1 of 4 groups to receive either BIIB115 or a placebo. A placebo looks like the study drug but contains no real medicine.
* Participants will receive a single dose of either BIIB115 or the placebo as an injection directly into the spinal canal on Day 1.
* Neither the researchers nor the participants will know if the participants will receive BIIB115 or the placebo.
* The treatment and follow up period will last for 13 months.
* Participants will have up to 6 clinic visits and 4 telephone calls.
During Part B:
* In Part B, children with SMA will receive BIIB115. Both researchers and participants will know they are receiving BIIB115.
* Participants will receive 2 total doses of BIIB115 given at 2 different times.
* The treatment and follow up period will last for 25 months.
* Participants will have up to 14 clinic visits and 6 telephone calls.
In both parts, participants will stay in the clinic for 24 hours after each dose for so that researchers can check on their health and any medical problems they might have.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:6 - 55
Sex:All
62 Participants Needed
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My name is Bask, and I helped to start the company here. We started Power when my Dad was diagnosed with multiple myeloma, and I needed a better way to understand how he could access the most promising immunotherapy for his illness.
Bask GillCEO at Power
Frequently Asked Questions
How much do Spinal Muscular Atrophy clinical trials in Miami, FL pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Spinal Muscular Atrophy clinical trials in Miami, FL work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Spinal Muscular Atrophy trials in Miami, FL 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length in Miami, FL for Spinal Muscular Atrophy is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility in Miami, FL several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Spinal Muscular Atrophy medical study in Miami, FL ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Spinal Muscular Atrophy clinical trials in Miami, FL ?
Most recently, we added Nusinersen for Spinal Muscular Atrophy, Exergaming for Spinal Muscular Atrophy and Risdiplam for Spinal Muscular Atrophy to the Power online platform.