Vimizim: Uses, Dosage & Side Effects
Enzyme replacement therapy for Morquio A syndrome (mucopolysaccharidosis type IVA), replacing the deficient N-acetylgalactosamine-6-sulfatase enzyme in both adults and children
Vimizim (elosulfase alfa) is an enzyme replacement therapy used to treat mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. This rare, inherited lysosomal storage disorder is caused by a deficiency of the enzyme N-acetylgalactosamine-6-sulfatase (GALNS), which leads to the accumulation of glycosaminoglycans, primarily keratan sulfate, in tissues throughout the body. Vimizim provides a recombinant form of the missing enzyme and has been shown to improve walking ability and reduce keratan sulfate levels. It is administered as a weekly intravenous infusion in both adults and children and is intended for lifelong treatment.
Quick Facts: Vimizim
Key Takeaways
- Vimizim (elosulfase alfa) is the only approved enzyme replacement therapy for mucopolysaccharidosis type IVA (Morquio A syndrome), replacing the deficient N-acetylgalactosamine-6-sulfatase (GALNS) enzyme needed to break down keratan sulfate and chondroitin-6-sulfate.
- Clinical trials demonstrated that Vimizim improves the 6-minute walk test distance and significantly reduces urinary keratan sulfate levels, indicating reduced glycosaminoglycan accumulation in tissues including cartilage and bone.
- The recommended dose is 2 mg/kg body weight administered once weekly as an intravenous infusion over approximately 4 hours; the same dose applies to both adults and children, and treatment should begin as early as possible.
- Infusion-associated reactions, including potentially severe anaphylaxis, are the most important safety concern; premedication with antihistamines is recommended before each infusion, and healthcare professionals should be prepared to manage allergic reactions.
- Vimizim is intended for lifelong treatment as it does not cure the underlying genetic defect; patients with spinal cord compression should be closely monitored, as this complication is part of the natural history of MPS IVA.
What Is Vimizim and What Is It Used For?
Vimizim contains the active substance elosulfase alfa, a recombinant (biotechnologically produced) form of the human enzyme N-acetylgalactosamine-6-sulfatase (GALNS). It belongs to a class of medications known as enzyme replacement therapies (ERTs), which are designed to supplement or replace enzymes that are absent or deficient in patients with lysosomal storage disorders. Vimizim is specifically approved for the treatment of mucopolysaccharidosis type IVA (MPS IVA) in both adults and children of all ages.
Mucopolysaccharidosis type IVA, commonly known as Morquio A syndrome, is a rare, autosomal recessive genetic disorder. It is caused by mutations in the GALNS gene, which encodes the lysosomal enzyme N-acetylgalactosamine-6-sulfatase. This enzyme is essential for the normal breakdown (catabolism) of certain glycosaminoglycans (GAGs), specifically keratan sulfate (KS) and chondroitin-6-sulfate (C6S). These GAGs are complex sugar molecules found abundantly in connective tissues, particularly in cartilage, bone, cornea, and heart valves.
In individuals with MPS IVA, the complete absence or severe deficiency of GALNS means that keratan sulfate and chondroitin-6-sulfate cannot be properly degraded within the lysosomes of cells. As a result, these undegraded substrates progressively accumulate in tissues throughout the body, disrupting their normal structure and function. The clinical consequences of this accumulation are wide-ranging and typically become apparent in early childhood, often between the ages of 1 and 3 years.
The hallmark features of Morquio A syndrome include progressive skeletal abnormalities (skeletal dysplasia), short stature, joint hypermobility with progressive joint stiffness, genu valgum (knock-knees), pectus carinatum (prominent breastbone), and spinal abnormalities including odontoid hypoplasia, which can lead to atlantoaxial instability and spinal cord compression. Beyond the skeleton, patients may experience corneal clouding, hearing loss, dental abnormalities (thin enamel), hepatomegaly, and cardiac valve disease. Respiratory compromise due to restrictive lung disease and airway obstruction is a major cause of morbidity and mortality. The clinical severity of MPS IVA varies widely, from severe forms with onset in infancy to attenuated forms that may not be diagnosed until adulthood.
How Vimizim Works
Vimizim works by providing a functional copy of the enzyme that patients with MPS IVA lack. When administered intravenously, elosulfase alfa is distributed throughout the body and taken up by cells via mannose-6-phosphate receptors on their surface. Once inside the cell, the enzyme is directed to the lysosomes, the cellular compartments where glycosaminoglycan degradation normally occurs. Within the lysosomes, elosulfase alfa catalyzes the removal of sulfate groups from the non-reducing terminal N-acetylgalactosamine-6-sulfate residues of keratan sulfate and chondroitin-6-sulfate, thereby enabling their further degradation and preventing continued accumulation.
Clinical trials have demonstrated that Vimizim treatment leads to measurable improvements in endurance and mobility, as assessed by the 6-minute walk test (6MWT). In the pivotal MOR-004 phase 3 trial, patients receiving Vimizim at the recommended dose of 2 mg/kg weekly showed a statistically significant improvement in the 6MWT distance compared with placebo over 24 weeks. Additionally, treatment resulted in a substantial reduction in urinary keratan sulfate levels, a key biomarker of the disease, confirming that the exogenous enzyme was reaching its target and reducing substrate accumulation.
MPS IVA (Morquio A syndrome) is an ultra-rare condition with an estimated prevalence of approximately 1 in 200,000 to 1 in 300,000 live births worldwide, though higher prevalences have been reported in certain populations. Vimizim was granted orphan drug designation by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), recognizing the unmet medical need for this patient population. It received its first regulatory approval in 2014.
What Should You Know Before Taking Vimizim?
Contraindications
Vimizim is contraindicated in patients who have experienced life-threatening (anaphylactic) allergic reactions to elosulfase alfa or to any of the inactive ingredients in the formulation, and for whom re-challenge with the medication cannot be adequately managed. Anaphylaxis is a serious, potentially fatal allergic reaction that requires immediate medical intervention. If a patient has previously experienced anaphylaxis during Vimizim infusion, the decision to re-challenge must be made carefully by a specialist physician, with appropriate resuscitation equipment and trained staff immediately available.
The excipients in Vimizim include sodium acetate trihydrate, monosodium phosphate monohydrate, arginine hydrochloride, sorbitol, polysorbate 20, and water for injection. Patients with known hypersensitivity to any of these components should not receive Vimizim. In particular, patients with hereditary fructose intolerance (HFI) should not receive this medication because it contains sorbitol (100 mg per 5 ml vial, equivalent to 40 mg/kg). Sorbitol is a source of fructose, and patients with HFI cannot metabolize fructose, which can lead to serious adverse effects including hypoglycemia, hepatic failure, and metabolic acidosis.
Warnings and Precautions
Infusion-associated reactions (IARs) are the most commonly reported adverse events with Vimizim and can occur during or within 24 hours after the infusion. These reactions range from mild symptoms such as headache, nausea, and fever to severe and potentially life-threatening anaphylaxis. In clinical trials, IARs were reported in a substantial proportion of patients, and some reactions required medical intervention including slowing or temporarily stopping the infusion, and administering additional medications such as antihistamines, corticosteroids, or antipyretics.
To reduce the risk of infusion-associated reactions, it is recommended that patients receive premedication with antihistamines (with or without antipyretics) approximately 30 to 60 minutes prior to each infusion. The decision to administer premedication and the choice of agents should be guided by the treating physician, taking into account the patient's history of prior reactions. If a reaction occurs during the infusion, the healthcare provider may slow the infusion rate or temporarily stop the infusion, administer additional antihistamines or corticosteroids, and resume the infusion at a lower rate once symptoms have resolved.
Patients with MPS IVA are at risk of spinal cord compression due to the progressive nature of the skeletal disease, particularly atlantoaxial instability and cervical stenosis caused by odontoid hypoplasia. Symptoms of spinal cord compression may include back pain, numbness or weakness in the arms or legs, and loss of bladder or bowel control. These symptoms may be part of the underlying disease process and may occur regardless of Vimizim treatment. Patients should be instructed to report any new neurological symptoms immediately, and regular monitoring of spinal integrity is recommended.
If you develop back pain, numbness in your arms or legs, or difficulty controlling urination or bowel movements, contact your doctor immediately. These symptoms may indicate spinal cord compression, a known complication of MPS IVA that requires urgent medical evaluation and possible surgical intervention.
Pregnancy and Breastfeeding
There are limited data on the use of Vimizim in pregnant women. Animal studies with elosulfase alfa have not shown any adverse effects on fertility or fetal development; however, the absence of risk in animal studies does not guarantee safety in humans. As a precaution, Vimizim should not be used during pregnancy unless clearly necessary and the potential benefit to the mother outweighs the potential risk to the fetus. Women of childbearing potential should discuss family planning with their healthcare provider before starting treatment.
It is not known whether elosulfase alfa is excreted in human breast milk. Because many drugs are excreted in human milk, the potential benefits of breastfeeding should be weighed against the potential risk to the infant. The treating physician should help the mother make an informed decision based on the importance of Vimizim treatment for the mother's health and any potential effects on the nursing infant.
The effects of Vimizim on human fertility are not known. In preclinical studies, no adverse effects on fertility were observed in animals treated with elosulfase alfa at doses up to several times the recommended human dose, adjusted for body surface area.
Driving and Operating Machinery
Dizziness has been reported in some patients during or after Vimizim infusion. Patients who experience dizziness should be advised not to drive vehicles or operate machinery until the symptoms have resolved. This is particularly important on infusion days, and patients should plan accordingly.
Sodium and Sorbitol Content
Each 5 ml vial of Vimizim contains 8 mg of sodium, equivalent to 0.4% of the WHO-recommended maximum daily intake of 2 g of sodium for an adult. This should be taken into account for patients on a controlled sodium diet. Additionally, each 5 ml vial contains 100 mg of sorbitol (E420). As noted above, patients with hereditary fructose intolerance must not receive this medication.
How Does Vimizim Interact with Other Drugs?
Elosulfase alfa is a recombinant protein that is metabolized through normal protein degradation pathways within cells. It does not interact with the cytochrome P450 enzyme system or other common drug-metabolizing pathways. Therefore, pharmacokinetic interactions with other medications metabolized through these systems are considered unlikely. No formal drug interaction studies have been performed with Vimizim.
Although no specific drug interactions have been identified, it is important for patients to inform their healthcare provider about all medications they are currently taking, have recently taken, or may be planning to take. This includes prescription medications, over-the-counter drugs, vitamins, and herbal supplements. This comprehensive medication review allows the treating physician to monitor for any unexpected interactions and adjust treatment plans as needed.
Vimizim must not be mixed with other medicinal products in the same infusion line, except for sodium chloride 9 mg/ml (0.9%) solution, which is used as the diluent for preparing the infusion. Using other diluents or mixing Vimizim with other drugs could potentially affect the stability, efficacy, or safety of the enzyme.
Premedication Considerations
Patients routinely receive premedication with antihistamines (and sometimes corticosteroids or antipyretics) before each Vimizim infusion to reduce the risk of infusion-associated reactions. These premedications are well-established in clinical practice and are not considered to interact with elosulfase alfa in a clinically significant way. The choice of antihistamine (e.g., cetirizine, diphenhydramine) and whether to include a corticosteroid or antipyretic should be individualized based on the patient's medical history and prior reaction profile.
Immunological Considerations
Most patients treated with Vimizim develop anti-drug antibodies (ADAs) to elosulfase alfa over time. In clinical trials, the majority of patients had developed ADAs by week 4 of treatment. The clinical significance of these antibodies is still being studied, but in some patients, high antibody titers have been associated with a reduced treatment response and an increased risk of infusion-associated reactions. There is no established interaction between the development of ADAs and the use of other medications, but patients receiving immunosuppressive therapies may have a different antibody response profile.
What Is the Correct Dosage of Vimizim?
Vimizim is administered exclusively by intravenous infusion under the supervision of a healthcare professional experienced in the management of MPS IVA or other lysosomal storage disorders. It is not available as an oral medication, and self-administration at home is generally not recommended during the initial treatment period, though home infusion programs may be available in some regions after a period of successful hospital-based treatment.
Adults
Adult Dosage
The recommended dose for adults is 2 mg/kg body weight administered once weekly as an intravenous infusion. The total infusion volume depends on the patient's weight: patients weighing 25 kg or more receive a total infusion volume of 250 ml (diluted in 0.9% sodium chloride solution). Each infusion takes approximately 3.5 to 4 hours to complete, including the gradual escalation of the infusion rate.
Children
Pediatric Dosage
The dose for children is the same as for adults: 2 mg/kg body weight once weekly by intravenous infusion. For patients weighing less than 25 kg, the total infusion volume is 100 ml (diluted in 0.9% sodium chloride solution). Treatment can be initiated at as early an age as possible, as the progressive nature of MPS IVA means that early intervention may help preserve physical function. Clinical data are available for children as young as 5 years from the pivotal trials, and post-marketing experience includes younger patients.
Elderly
Elderly Dosage
There are limited clinical data on the use of Vimizim in patients over 65 years of age, as MPS IVA is typically diagnosed in childhood. No specific dose adjustment is recommended for elderly patients. The standard dose of 2 mg/kg once weekly should be used, with appropriate monitoring as for any other patient.
Infusion Rate Schedule
The infusion rate of Vimizim is gradually increased during each infusion to minimize the risk of infusion-associated reactions. The infusion rate schedule differs based on the patient's weight and corresponding total infusion volume.
| Patient Weight | Infusion Volume | Initial Rate (0–15 min) | Step 2 (15–30 min) | Step 3 (30–45 min) | Max Rate (90+ min) |
|---|---|---|---|---|---|
| < 25 kg | 100 ml | 3 ml/h | 6 ml/h | 12 ml/h | 36 ml/h |
| ≥ 25 kg | 250 ml | 6 ml/h | 12 ml/h | 24 ml/h | 72 ml/h |
The infusion rate is increased every 15 minutes in a stepwise manner as tolerated by the patient. If an infusion-associated reaction occurs, the rate may be slowed or the infusion temporarily paused. The treating physician may adjust the rate escalation schedule based on the individual patient's tolerance.
Missed Dose
If a Vimizim infusion is missed, patients should contact their healthcare provider to reschedule the infusion as soon as possible. Consistent weekly dosing is important to maintain the therapeutic benefit of enzyme replacement therapy. Patients should not receive a double dose to compensate for a missed infusion. The regular weekly infusion schedule should be resumed at the earliest opportunity.
Overdose
There is limited experience with overdose of Vimizim in clinical studies. No specific overdose symptoms have been reported. In the event of an overdose, patients should be closely monitored and receive supportive care as needed. There is no specific antidote for elosulfase alfa overdose. If overdose is suspected, the infusion should be stopped and the patient monitored for any signs of adverse reactions, particularly infusion-associated reactions.
Vimizim must be prepared by a healthcare professional using aseptic technique. Each single-use vial should be allowed to reach room temperature (23–27°C) before use. The concentrate is diluted with 0.9% sodium chloride solution to the appropriate volume (100 ml or 250 ml depending on patient weight). The diluted solution should be inspected visually for particulate matter and discoloration; do not use if the solution appears discolored or contains visible particles. An in-line filter of 0.2 μm may be used during infusion.
What Are the Side Effects of Vimizim?
Like all medicines, Vimizim can cause side effects, although not everyone will experience them. The majority of adverse reactions reported with Vimizim are infusion-associated reactions (IARs) that occur during the infusion or within 24 hours afterward. The severity of these reactions ranges from mild and self-limiting to severe and potentially life-threatening. In clinical trials, IARs were the most frequently reported adverse events and were observed in a significant proportion of patients.
Healthcare professionals administering Vimizim should be prepared to manage infusion-associated reactions, including anaphylaxis. Appropriate medical support measures, including resuscitation equipment and trained staff, should be readily available during each infusion. Patients should be observed for at least 60 minutes after the completion of each infusion, particularly during the early weeks of treatment when the risk of reactions may be higher.
Very Common
May affect more than 1 in 10 people
- Headache
- Nausea
- Vomiting
- Fever (pyrexia)
- Chills
- Abdominal pain
- Diarrhea
- Oropharyngeal pain (sore throat)
- Dizziness
- Dyspnea (difficulty breathing)
Common
May affect up to 1 in 10 people
- Myalgia (muscle pain)
- Rash, itching, or urticaria (hives)
Uncommon
May affect up to 1 in 100 people
- Anaphylaxis (severe allergic reaction)
- Hypotension (low blood pressure)
Anaphylaxis is the most serious potential side effect of Vimizim. Symptoms of a severe allergic reaction may include difficulty swallowing, severe shortness of breath or wheezing, swelling of the face or lips, dizziness, or a weak pulse. If any of these symptoms occur during or after an infusion, immediate medical treatment is required. Depending on the severity of the reaction, the healthcare provider may slow down, temporarily stop, or permanently discontinue the infusion and administer rescue medications such as epinephrine, antihistamines, or corticosteroids.
Mild to moderate infusion-associated reactions are generally manageable and do not necessitate permanent discontinuation of treatment. The rate of IARs tends to be highest during the initial weeks of treatment and may decrease over time as patients develop tolerance to the infusion. However, reactions can occur at any point during the course of treatment, and vigilance should be maintained throughout.
Seek emergency medical attention if you experience difficulty breathing, severe wheezing, swelling of the face, lips, or throat, a rapid heartbeat with dizziness, or a widespread skin rash with itching during or after a Vimizim infusion. These may be signs of a serious allergic reaction (anaphylaxis) that requires immediate treatment.
Reporting Side Effects
Reporting suspected side effects after a medicine has been authorized is important for the ongoing monitoring of its benefit–risk balance. Healthcare professionals and patients are encouraged to report any suspected adverse reactions to their national pharmacovigilance authority. In the EU, reports can be submitted through the national reporting systems listed on the EMA website. In the US, reports should be submitted to the FDA MedWatch program.
How Should You Store Vimizim?
Proper storage of Vimizim is essential to maintain the integrity and efficacy of the medication. Unopened vials should be stored in a refrigerator at a temperature of 2°C to 8°C (36°F to 46°F). The vials must not be frozen at any time, as freezing can damage the protein structure of elosulfase alfa and render the medication ineffective or unsafe. The vials should be kept in their original outer packaging to protect the concentrate from light exposure, which could also degrade the active substance.
Before use, each vial should be visually inspected. The concentrate should appear clear to slightly opalescent and colorless to pale yellow. It should be free of visible particles. If the solution appears discolored, turbid, or contains visible particulate matter, the vial should not be used and should be disposed of appropriately.
Once diluted for infusion, the Vimizim solution should ideally be used immediately. If immediate use is not possible, the diluted solution may be stored at 2°C to 8°C for a maximum of 24 hours, followed by up to an additional 24 hours at room temperature (23°C to 27°C) during the infusion itself. Any unused portion of the diluted solution should be discarded and not saved for later use, as the product does not contain preservatives.
Keep Vimizim out of the sight and reach of children. Do not use the medication after the expiration date printed on the carton and vial label. The expiration date refers to the last day of the stated month. Unused or expired medication should not be disposed of via household waste or wastewater. Consult your pharmacist or local waste disposal guidelines for proper disposal methods to protect the environment.
What Does Vimizim Contain?
Vimizim is supplied as a concentrate for solution for infusion. Each single-use vial contains 5 ml of concentrate at a concentration of 1 mg/ml, providing a total of 5 mg of elosulfase alfa per vial. Elosulfase alfa is a recombinant form of human N-acetylgalactosamine-6-sulfatase (GALNS) produced by recombinant DNA technology in Chinese hamster ovary (CHO) cell lines. The enzyme is a glycoprotein with mannose-6-phosphate residues that facilitate its uptake into target cells via the mannose-6-phosphate receptor pathway.
| Component | Type | Amount per Vial |
|---|---|---|
| Elosulfase alfa | Active ingredient | 5 mg (1 mg/ml) |
| Sodium acetate trihydrate | Buffer | Quantity sufficient |
| Monosodium phosphate monohydrate | Buffer | Quantity sufficient |
| Arginine hydrochloride | Stabilizer | Quantity sufficient |
| Sorbitol (E420) | Stabilizer / tonicity agent | 100 mg |
| Polysorbate 20 | Surfactant | Quantity sufficient |
| Water for injection | Solvent | To 5 ml |
The concentrate appears as a clear to slightly opalescent and colorless to pale yellow liquid. Each vial is intended for single use only and should not be re-used or stored after opening. The packaging size is 1 vial per carton.
Vimizim is manufactured by BioMarin International Limited, based in Shanbally, Ringaskiddy, County Cork, Ireland. The marketing authorization holder is also BioMarin International Limited.
Frequently Asked Questions About Vimizim
Morquio A syndrome (MPS IVA) is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme N-acetylgalactosamine-6-sulfatase (GALNS). Without this enzyme, the body cannot properly break down certain complex sugar molecules called glycosaminoglycans (particularly keratan sulfate). These molecules accumulate in tissues throughout the body, especially cartilage and bone, causing progressive skeletal abnormalities, short stature, joint problems, corneal clouding, hearing loss, and respiratory difficulties. Enzyme replacement therapy with Vimizim provides a functional copy of the missing enzyme to help reduce this accumulation.
Vimizim is administered once weekly as an intravenous infusion. Each infusion takes approximately 4 hours to complete due to the gradual escalation of the infusion rate. The infusions are given in a hospital or clinic setting by a trained healthcare professional. Since MPS IVA is a chronic, lifelong condition, Vimizim treatment is intended to continue indefinitely. Some patients may eventually be eligible for home infusion programs after demonstrating tolerance in a clinical setting.
Vimizim can reduce the ongoing accumulation of keratan sulfate and has been shown to improve walking ability (as measured by the 6-minute walk test). However, it cannot reverse skeletal damage that has already occurred, such as bone deformities, spinal abnormalities, or joint damage. This is why early diagnosis and initiation of treatment are crucial—starting enzyme replacement therapy before irreversible damage occurs may help preserve physical function. Many patients still require orthopedic interventions and other supportive treatments alongside enzyme replacement therapy.
If you experience any symptoms during a Vimizim infusion—such as headache, nausea, fever, chills, rash, itching, difficulty breathing, or dizziness—inform the healthcare professional administering the infusion immediately. They may slow down or temporarily pause the infusion and provide additional medications (such as antihistamines or corticosteroids) to manage the reaction. In most cases, mild to moderate reactions can be managed without permanently stopping treatment. For severe allergic reactions (anaphylaxis), emergency treatment including epinephrine will be administered.
Yes, Vimizim is approved for use in patients of all ages, including young children. Early initiation of treatment is recommended because MPS IVA is a progressive disease, and starting enzyme replacement therapy before significant tissue damage occurs may help preserve function. The dose (2 mg/kg weekly) is the same for children and adults, with the infusion volume adjusted based on the child's weight. Pediatric patients should be closely monitored during infusions for any signs of infusion-associated reactions, and premedication is recommended as for adult patients.
Currently, Vimizim is the only approved enzyme replacement therapy for MPS IVA. Management of Morquio A syndrome also involves a multidisciplinary approach including orthopedic surgery (for spinal stabilization and joint corrections), cardiac monitoring, ophthalmological assessments, hearing aids, pulmonary support, and physiotherapy. Gene therapy and substrate reduction therapy are being investigated in clinical trials as potential future treatments, but none are currently approved. Hematopoietic stem cell transplantation has not shown consistent benefit in MPS IVA, unlike some other mucopolysaccharidoses.
References
- European Medicines Agency. Vimizim (elosulfase alfa) – Summary of Product Characteristics. Available at: EMA EPAR Vimizim. Accessed February 2026.
- U.S. Food and Drug Administration. Vimizim (elosulfase alfa) – Prescribing Information. Approved February 2014. Available at: FDA Prescribing Information. Accessed February 2026.
- Hendriksz CJ, Burton B, Fleming TR, et al. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014;37(6):979–990. doi:10.1007/s10545-014-9715-6
- Hendriksz CJ, Berger KI, Giugliani R, et al. International guidelines for the management and treatment of Morquio A syndrome. Am J Med Genet A. 2015;167A(1):11–25. doi:10.1002/ajmg.a.36833
- Tomatsu S, Montano AM, Oikawa H, et al. Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment. Curr Pharm Biotechnol. 2011;12(6):931–945. doi:10.2174/138920111795542615
- World Health Organization. WHO Model List of Essential Medicines. 23rd List, 2023. Available at: WHO Essential Medicines.
- Harmatz P, Mengel KE, Giugliani R, et al. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. Mol Genet Metab. 2013;109(1):54–61. doi:10.1016/j.ymgme.2013.01.021
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