Referral and enrollment forms

ADSTILADRIN® is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

Enrollment form

Bylvay® is an ileal bile acid transporter (IBAT) inhibitor indicated for: Progressive Familial Intrahepatic Cholestasis (PFIC) the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC).  Alagille Syndrome (ALGS) the treatment of cholestatic pruritus in patients 12 months of age and older with Alagille syndrome (ALGS).

Enrollment and consent form

ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age: For the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the DMD gene. For the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro[1]dystrophin (noted hereafter as “micro-dystrophin”). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

English form

Spanish form

FILSPARI is an endothelin and angiotensin II receptor antagonist indicated to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.

Enrollment form

KUVAN® is a phenylalanine hydroxylase activator indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN® is to be used in conjunction with a Phe-restricted diet.

Enrollment and consent form

LUMRYZ is a central nervous system depressant indicated for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.

Enrollment form

NITYR® is a hydroxyphenyl-pyruvate dioxygenase inhibitor used to treat adults and children with hereditary tyrosinemia type 1 (HT-1). NITYR works by preventing the body from breaking down an amino acid called tyrosine and by keeping other toxic substances from building up and causing harm to liver or kidneys.

Enrollment form

PALYNZIQ® is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe concentrations in adult patients with phenylketonuria who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.

Enrollment and consent form

QALSODY™ (tofersen) is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Enrollment form

REBYOTA® is a novel first-in-class microbiota-based live biotherapeutic indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI.

Enrollment form

RYONCIL is an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.

Enrollment form

Sapropterin is a generic and is a phenylalanine hydroxylase activator indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Sapropterin is to be used in conjunction with a Phe-restricted diet.

No form currently

SEPHIENCE™ is a phenylalanine hydroxylase (PAH) activator indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE™ is to be used in conjunction with a phenylalanine (Phe)-restricted diet.

Enrollment form

Sucraid® is an enzyme replacement therapy for the treatment of genetically determined sucrase deficiency, which is part of Congenital Sucrase-Isomaltase Deficiency (CSID).

Enrollment form

VOXZOGO® is a C type natriuretic peptide (CNP) analog indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Enrollment form