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What is Aldurazyme used for?

What is Aldurazyme used for?

Aldurazyme (laronidase) is a medication used to treat symptoms of certain forms of a rare, genetic disease called mucopolysaccharidosis type I (MPS I).

How do you administer Aldurazyme?

The recommended dosage regimen of ALDURAZYME is 0.58 mg/kg of body weight administered once weekly as an intravenous (IV) infusion. Pretreatment with antipyretics and/or antihistamines is recommended 60 minutes prior to the start of the infusion [see Warnings and Precautions (5)].

Who manufactures ALDURAZYME?

Under the revised structure, the operational responsibilities for BioMarin and Genzyme will not significantly change. Genzyme will continue to globally market and sell Aldurazyme for mucopolysaccharidosis I (MPS I) and BioMarin will continue to manufacture Aldurazyme.

How do you mix Aldurazyme?

Withdraw the required volume from the Aldurazyme vials and combine the withdrawn volumes. Add the combined volumes of Aldurazyme to the sodium chloride 9 mg/ml (0.9%) solution for infusion. Mix the solution for infusion gently. Prior to use visually inspect the solution for particulate matter.

How much does Aldurazyme cost?

Aldurazyme (laronidase), for Hurler syndrome (mucopolysaccharidosis I), annual cost: $200,000.

How is enzyme replacement therapy done?

Enzyme Replacement Therapy: The Basics The most common method of ERT is through IV infusions, in which the replacement enzyme is administered directly into the bloodstream through a controlled drip of fluids.

What is Hurler syndrome?

Hurler syndrome is an inherited condition caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. As a result, undigested sugar molecules build up in the body, causing progressive damage to the brain, heart, and other organs.

Which is the costliest drug in the world?

It’s Zolgensma (generic name: onasemnogene abeparvovec-xioi), a life-saving gene therapy approved in May 2019 to treat pediatric Spinal Muscular Atrophy (SMA). Zolgensma’s total cost is $2.125 million, according to Novartis, the manufacturer. It’s considered the world’s most expensive single-dose drug.

Which is the most costly medicine in the world?

The most recent treatment is Zolgensma (generic name onasemnogene abeparvovec), a pioneering gene therapy dubbed “the most expensive drug in the world” and only available through the NHS since March 2021. Zolgensma uses a harmless virus with some of its DNA replaced by a copy of the human SMN1 gene.

What are the disadvantages of enzyme replacement therapy?

However, there are certain disadvantages: Not all patients are suited for this treatment. Some organs and tissues are treated more readily than others and there are problems with gauging efficacy. They are expensive, thereby limiting access to patients.

What are the side effects of enzyme replacement therapy?

As with any medication, enzyme replacement therapy may have side effects. These include irritation at the site where the IV is inserted, electrolyte imbalance, fever, or an allergic reaction. Additional symptoms may include headaches, tremors, chest tightness, nausea, and/or vomiting.

How is Hurler diagnosed?

What are the symptoms of Hurler syndrome?

  1. clouding of the front part of the eye (corneal clouding)
  2. frequent upper respiratory infections.
  3. enlarged tonsils and/or adenoids.
  4. distinct facial features (coarse facial features)
  5. hernias.

What is the difference between Hunter and Hurler syndrome?

Hunter syndrome (MPS II) is distinguished from Hurler syndrome by an X-linked recessive inheritance, longer survival, lack of corneal clouding, and the different biochemical defect with deficiency of the lysosomal enzyme iduronate-2-sulfatase. As with Hurler syndrome, patients show coarse, straight scalp hair.

Which country has the most expensive medicine?

The United States
The United States: the world’s highest medical expenses The United States has the most expensive healthcare system of any country. A medical consultation with a general practitioner costs, on average, $190 or around €170. A stay in hospital can result in bills amounting to tens or even hundreds of thousands of dollars.

Do doctors get compensated for writing prescriptions?

Under this statute, it is illegal for a physician to receive remuneration for referring a patient for a service that will be paid in whole or in part by a federal health care program or for prescribing or recommending the purchase of a drug that will be paid in whole or in part by a federal health care program.

Which animal provides highest medicine?

Answer: Plants serve as the source of the highest medicines derived worldwide.

How much does enzyme replacement cost?

Enzyme replacement therapy (ERT) resulted in 4.21038 quality-adjusted life years (QALY) per $381,852. The incremental cost per QALY was $96,809 and the incremental cost per life years gained (LYG) was 74,429 over a 22-year time horizon. Sensitivity analysis indicated the robustness of the results.

How often is enzyme replacement therapy done?

ERT is administered by IV infusion. Typically, infusions occur every week or every two weeks. For some types of ERT, these infusions can occur as infrequently as every four weeks.

Which diseases can be treated with enzyme therapy?

The most common conditions treated by ERT are lysosomal storage diseases (LSDs)….Pompe Disease

  • Poor muscle development.
  • Trouble eating, breathing, and/or hearing.
  • Respiratory problems.
  • Enlarged liver and/or heart.
  • Problems with motor function.

What is a limitation of enzyme replacement therapy?

Not all patients are suitable for treatment, some organs and tissues are corrected more readily than others, and there are problems with gauging efficacy in these highly variable disorders.

How is Aldurazyme administered?

The recommended dosage regimen of Aldurazyme is 0.58 mg/kg of body weight administered once weekly as an intravenous infusion. Pretreatment is recommended 60 minutes prior to the start of the infusion and may include antihistamines, antipyretics, or both [ see Warnings and Precautions (5) ].

What is the pH of Aldurazyme?

Aldurazyme, for intravenous infusion, is supplied as a sterile, nonpyrogenic, colorless to pale yellow, clear to slightly opalescent solution that must be diluted prior to administration in 0.9% Sodium Chloride Injection, USP. The solution in each vial contains a nominal laronidase concentration of 0.58 mg/mL and a pH of approximately 5.5.

What are the benefits of Aldurazyme?

ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder. Download full prescribing info including boxed warning.

How common are allergic reactions to Aldurazyme?

In clinical studies and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious allergic reactions. In patients with MPS I, pre-existing upper airway obstruction may have contributed to the severity of some reactions.

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