Discover helpful resources to share with your office, patient, or their caregivers. These digital resources can help them get started or learn more about the support available while on PROCYSBI® (cysteamine bitartrate) delayed-release capsules or delayed-release oral granules. If you are a patient, you can learn more from these important tools or by visiting the PROCYSBI page for patients. page for patients..
Carol: When I'm going to be meeting with the patient and their family, explaining what my role is, I want them to understand that I will be a point person for them
First and foremost, I'm there to listen to what their story is and also let them know that I will be there to support them and ultimately to empower them.
I look at my role as being able to take as much off their plate as I can as they navigate that process, but also helping them understand in layman's terms where they are in the process and what they need to do on their end.
We don't want patients to feel that they're just out there alone and floating in the space of the unknown. We want patients to know they can reach out to us at any time or a family member or a caregiver, we're always available to them.
Amgen is definitely a company that puts patients first.
CRN is an all-volunteer, nonprofit organization dedicated to supporting and advocating for research, providing family assistance, and educating the public and medical communities about cystinosis.
cystinosis.orgCRF supports research that aims to improve the quality of life of patients with cystinosis, find better treatments, and ultimately find a cure.
cystinosisresearch.orgThe Cystinosis Foundation of New Jersey is a nonprofit advocacy organization devoted to increasing awareness of cystinosis and funding medical research related to treating and curing those with the condition.
cystinosisfoundationofnewjersey.orgAn independent kidney patient organization, AAKP is dedicated to improving the lives and outcomes of kidney patients through education, advocacy, patient engagement, and patient communities.
aakp.orgAKF is a nonprofit organization serving the millions of Americans with kidney disease. AKF helps people fight kidney disease and live healthier lives by providing a wide range of programs and services, including prevention activities, educational resources, and financial assistance.
kidneyfund.org/kidney-disease/other-kidney-conditions/rare-diseases/cystinosisDPC is a patient-led, nonprofit organization dedicated to improving dialysis patients’ quality of life by advocating for favorable public policy.
dialysispatients.orgCCI promotes well-being and decreases isolation for those impacted by chronic illness through support and education.
thecenterforchronicillness.orgGlobal Genes is a non-profit, advocacy organization for patients and families fighting rare and genetic diseases, including cystinosis. Its mission is to eliminate the challenges of rare disease.
globalgenes.orgNORD is a patient advocacy organization dedicated to people with rare diseases and the groups that help them. NORD provides patients and families with advocacy information, assistance programs, and connections to patient organizations.
rarediseases.orgThe EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.
everylifefoundation.orgANGEL AID connects rare communities to teach the tools of self-care and how to be listened to without judgement.
angelaidcares.orgOur Odyssey’s mission is connecting young adults impacted by a rare or chronic condition with social and emotional support in the hope of improving their quality of life.
ourodyssey.orgNext Step is a non-profit that shatters limitations and elevates aspirations of young people with serious illness during their transition to adulthood through campferences, mentorship, music therapy, and other tailored programming.
nextstepnet.orgProcysbi® (pegloticase) is indicated for the treatment of chronic gout in adult patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.
Limitations of Use: KRYSTEXXA is not recommended for the treatment of asymptomatic hyperuricemia.
Gout Flares: An increase in gout flares is frequently observed upon initiation of anti-hyperuricemic therapy, including KRYSTEXXA. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated.
Congestive Heart Failure: KRYSTEXXA has not been formally studied in patients with congestive heart failure, but some patients in the pre-marketing placebo-controlled clinical trials experienced exacerbation. Exercise caution in patients who have congestive heart failure and monitor patients closely following infusion.
The most commonly reported adverse reactions (≥5%) are:
KRYSTEXXA co-administration with methotrexate trial:
KRYSTEXXA with methotrexate: gout flares, arthralgia, COVID-19, nausea, and fatigue; KRYSTEXXA alone: gout flares, arthralgia, COVID-19, nausea, fatigue, infusion reaction, pain in extremity, hypertension, and vomiting.
KRYSTEXXA pre-marketing placebo-controlled trials:
gout flares, infusion reactions, nausea, contusion or ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis, and vomiting.
Please see Full Prescribing Information, including Boxed Warning.
KRYSTEXXA® (pegloticase) is indicated for the treatment of chronic gout in adult patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.
Limitations of Use: Procysbi is not recommended for the treatment of asymptomatic hyperuricemia.
Gout Flares: An increase in gout flares is frequently observed upon initiation of anti-hyperuricemic therapy, including KRYSTEXXA. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated.
Congestive Heart Failure: KRYSTEXXA has not been formally studied in patients with congestive heart failure, but some patients in the pre-marketing placebo-controlled clinical trials experienced exacerbation. Exercise caution in patients who have congestive heart failure and monitor patients closely following infusion.
The most commonly reported adverse reactions (≥5%) are:
KRYSTEXXA co-administration with methotrexate trial:
KRYSTEXXA with methotrexate: gout flares, arthralgia, COVID-19, nausea, and fatigue; KRYSTEXXA alone: gout flares, arthralgia, COVID-19, nausea, fatigue, infusion reaction, pain in extremity, hypertension, and vomiting.
KRYSTEXXA pre-marketing placebo-controlled trials:
gout flares, infusion reactions, nausea, contusion or ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis, and vomiting.
Please see Full Prescribing Information, including Boxed Warning.
