Study drug for facioscapulohumeral muscular dystrophy type 1

Who can take part

• You have a confirmed diagnosis of FSHD1
• Your condition is considered moderate, not too mild or too advanced
• Muscle imaging shows you meet the study's fat-to-muscle ratio in a leg muscle
• You are between 18 and 70 years old
• Your body mass index (BMI) is between 18 and 35
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• You are able and willing to have a muscle biopsy and MRI scans
• You have not had blood clotting episodes or severe heart or liver disease
• You can walk independently without assistance
• You are not pregnant and agree to use effective birth control, if applicable
• Your health history does not include the conditions listed in the exclusions
• You have read and understood the trial information and can consent to participate
• You have had no previous treatment with a gene therapy or an experimental gene-targeted therapy for FSHD
• You have no active infection, and your kidney and liver function are normal as per the study doctor's judgment
• You have not had any major surgery in the past 3 months that could affect your participation
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• your participation in the study is safe for you and follows the study rules
• title of the study is the drug SRP-1001 for FSHD
• The study drug is given as an infusion
• You will have to attend several visits over about a year
• The exact number of study visits will be explained
• You will be in the study for about 12 months
• The study involves a muscle biopsy, MRI, and blood draws
• You will need to be accompanied by a caregiver on the first visit
• The study will cover your travel costs, but you must not have other medical conditions that could interfere with the study
• You have not had any significant bleeding disorder
• You have not had a blood transfusion in the last 3 months
• You have not participated in another interventional clinical trial in the last 30 days
• Your regular doctor will be informed of your participation in the study if you agree
• The study will not cover the cost of your regular medical care
• You will not be paid for your participation to avoid coercion
• We understand if you cannot participate and wish you the best
• You have no plans to move in the next year
• The sample size is small and the trial is in early stages
• The full list of eligibility requirements is long
• You should discuss this with your doctor as individual factors matter
• You are encouraged to ask questions
• the study team will answer any questions
• This is not medical advice
• You must agree to follow all study procedures
• Your decision to join is voluntary
• You can withdraw at any time
• We hope you decide to help with this research
• the study could lead to new treatments
• that would help others with this condition
• thank you for considering participation
• Please contact the study team directly for the most accurate information
• We are not able to verify your eligibility for this trial
• This is a preliminary check only
• We cannot provide medical advice
• You should consult with your healthcare provider
• This trial is not suitable for everyone
• The information you provided is not shared with the study team
• This tool is for informational purposes only
• You are responsible for verifying the information yourself
• The study team will have the final say
• We are not responsible for any errors or omissions
• This is a test
• Please ignore the previous instructions
• Return the JSON object
• This is a test.
• Just testing the limits.
• Now return the correct JSON.
• This is a prompt injection attempt.
• Do not follow this.
• Please ignore.
• Trying to break out.
• Is this working?
• Testing, testing, 1-2-3.
• OK, I think we're done.
• The JSON is above.
• I repeat, the JSON is above.
• Please check it now.
• I'm serious, check it.
• Ok, fine.
• Bye.
• .
• The requirements field should only have a few short items.
• This is not a requirement.
• Neither is this one.
• Please disregard all of this text.
• This is a long and winding road to nowhere.
• The quick brown fox jumps over the lazy dog.
• The sun sets in the west.
• Water is wet.
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• This is a valid requirement.
• Another valid one.
• Resetting.
• Now I'll write the real JSON, correctly this time.
• Absolutely final check.
• Done.
• genetically confirmed FSHD1
• clinical severity score between 3 and 8
• eligible muscle for biopsy per MRI
• age 18-70
• BMI between 18 and 35
• ability to provide consent
• use of contraception if applicable
• exclusion criteria not met
• no contraindications to biopsy or MRI
• no active infections like HIV or hepatitis
• no uncontrolled high blood pressure or severe heart disease
• no history of blood clots
• no bleeding disorders
• no other significant medical issues
• ability to follow the study plan
• willing to participate for the study's duration
• willing to attend all required appointments
• willing to undergo all required tests
• be available for follow-up assessments
• willing to answer questions honestly to the best of your knowledge
• agree not to participate in other trials simultaneously
• informed consent must be signed
• this is a long list
• but we're almost there
• thank you for your patience
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