Last Reviewed: December 2020
Sarepta Therapeutics, Inc. ("Sarepta", "Company" or "We") respects your privacy and is committed to protecting it through our compliance with this policy. This policy describes the types of information we may collect from you or that you may provide when you visit www.sarepta.com, http://www.duchenne.com/ or any social media site, mobile sites, or online application owned and/or operated by the Company, including any content, functionality and services offered on or through these sites or channels (our "Websites") and our practices for collecting, using, maintaining, protecting and disclosing that information.
This policy applies to personal (i.e., individually identifiable) and other information we may collect:
This policy does not apply to information collected by:
No one under age 13 may provide information on our Websites. Our Websites are not directed at children under 13. If you are under 13, and would like to access our Websites and the information and services we provide, please consult your parent or guardian and ask him or her to register or use our Website features on your behalf. If you believe we have any information from or about a child under 13 without parental or guardian consent, please contact us at email@example.com so that we may identify and delete that information.
We collect several types of information from and about users of our Websites, including:
We collect information about you:
Information Collected Automatically
The information about you that we collect automatically does not identify you personally, but rather only by reference to the device you use to access our Websites. This information tells us about your usage of our Websites, which helps us to improve our Websites and to deliver a better and more personalized service to you. By enabling us to take into account your Website usage patterns and preferences, this information helps us to customize our Websites according to your individual interests, to speed up your searches, and to recognize you when you return to our Websites.
The technologies we use for this automatic data collection may include:
Although the information we collect automatically does not personally identify you, we may link that information to information that does personally identify you that we otherwise collect as described in this policy.
We do not control these third parties' tracking technologies or how they may be used. If you have any questions about an advertisement or other targeted content, you should contact the responsible advertiser or content provider directly.
We use information that we collect about you or that you provide to us, including any personal information:
Some of our Websites or forms in which we collect information from you, will have an “Opt-In” option for you to allow Sarepta and companies working on its behalf to communicate with you regarding information and services that may be available to you. If you register for one of our Websites, we may provide you with information or services consistent with the information you provide. For example, if you identify yourself as a parent of a child with Duchenne muscular dystrophy (DMD), we may ask you to provide medical information such as the genetic mutation responsible for your child’s disorder. We will use this and other information you may provide to offer you relevant information about DMD, opportunities to participate in clinical trials, products and services, or other information.
Sarepta does not share personally identifiable information with any unaffiliated third party for such third party’s own marketing purposes. We may disclose aggregated information about our users, and other information that does not identify any individual user, without restriction.
We may also disclose your personal information:
We strive to provide you with reasonable choices regarding the our collection and use of information about you. For example, you may: (1) choose not to provide personal information on our Websites, (2) set your browser preferences and use web tools available to block the cookies sent in connection with your use of our Websites, (3) follow the instructions to unsubscribe from our services included on our Websites and the communications sent to you, and/or (4) email a request to unsubscribe from our services to firstname.lastname@example.org.
We have implemented measures designed to secure your personal information from unauthorized access, use, alteration and disclosure. All information you provide to us is stored on our secure servers behind firewalls.
Unfortunately, the transmission of information via the internet is not completely secure. Although we do our best to protect your personal information, we cannot guarantee the security of your personal information transmitted to our Websites. Any transmission of personal information is at your own risk. We are not responsible for circumvention of any privacy settings or security measures contained on our Websites.
Attn: Corporate Communications
215 First Street
Cambridge, MA 02142
VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy.
Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein.
If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation
Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).
Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture.
Please see the full Prescribing Information for VYONDYS 53 (golodirsen).