*Prices reflect U.S. average retail price

NITYR (nitisinone) tablets are a new option for treatment of hereditary tyrosinemia (HT-1).

Manufacturer's Website: Nityr                                                                                                                                 Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

Nitisinone (INN), also known as NTBC (an abbreviation of its full chemical name) is a medication used to slow the effects of hereditary tyrosinemia type 1. Since its first use for this indication in 1991, it has replaced liver transplantation as the first-line treatment for this rare condition... Wikipedia

Manufacturer's Website: Orfadin                                                                                                                                 Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

Metreleptin (trade name Myalept) is a synthetic analog of the hormone leptin used to treat diabetes and various forms of dyslipidemia. It has been approved in Japan for metabolic disorders including lipodystrophy and in the United States as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy... Wikipedia

Manufacturer's Website: Myalept                                                                                                                                Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

Asfotase alfa (trade name Strensiq) is a drug used in the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia... Wikipedia

Manufacturer's Website: Strensiq                                                                                                                                      Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are approved to reduce blood Phe levels in people with a certain type of Phenylketonuria (PKU). KUVAN is to be used with a Phe-restricted diet.

Manufacturer's Website: Kuvan                                                                                                                               Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

Eliglustat (INN, USAN; trade name Cerdelga) is a treatment for Gaucher's disease developed by Genzyme Corp that was approved by the FDA August 2014... Wikipedia

Manufacturer's Website: Cerdelga                                                                                                                   Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

Miglustat (OGT 918, N-butyl-deoxynojirimycin) is a drug developed by Oxford GlycoSciences and marketed by Actelion and is used primarily to treat type I Gaucher disease (GD1). It is marketed under the trade name Zavesca... Wikipedia

Manufacturer's Website: Zavesca                                                                                                                        Average retail cost: *

* This drug is only available at select specialty pharmacies, which usually means it may be expensive.

Vutrisiran, sold under the brand name Amvuttra, is a medication used for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is a small interfering RNA (siRNA) that interferes with the expression of the transthyretin (TTR) gene... Wikipedia

Manufacturer's Website: Amvuttra                                                                                                                                      Average retail cost: *

* This drug is administered by your healthcare practitioner and cannot be filled at a pharmacy. It may be expensive.