Rare disease

Phenylketonuria

Dihydrocetaxan

【Product name】Kuvan

[English common name]Sapropterin dihydrochloride

[Chinese common name]Dihydrocetaxan

[English name]SUN 0588, Sapropterin HCl, Sapropterin hydrochloride

[Chinese other name]

[Indications] Kuvan is suitable for treatmentPhenylketonuria(PKU) for all ages of adult and pediatric patientsHyperphenylalaninemia(HPA), these patients have been shown to respond to such treatments. Kuvan is also indicated for the treatment of hyperphenylalaninemia (HPA) in children and children of all ages with tetrahydrobiopterin (BH4) deficiency, which have been shown to respond to such treatment.

[Usage and Dosage] Kuvan is soluble in soluble tablets (100mg) or powder (100 or 500mg), soluble in water and consumed. Kuvan can only be obtained by prescription, and treatment must be initiated and supervised by a doctor with experience in treating PKU and BH4 deficiency. It is important that patients continue to take low phenylalanine and protein diets while taking Kuvan. The intake of phenylalanine and protein must be monitored and adjusted to ensure that blood phenylalanine levels and nutritional balance are controlled. Kuvan aims to be used for a long time.
The starting dose of Kuvan depends on the patient's weight. The dose is then adjusted according to the blood level of the amino acid including phenylalanine. Kuvan eats at the same time every day, preferably in the morning. For some patients with BH4 deficiency, it may be necessary to divide the dose into two or three doses in one day for best results.
A satisfactory response is defined as a decrease in blood phenylalanine levels of at least 30% or a level determined by a physician. If this goal is achieved after one month, the patient will be classified as a “responder” and will continue to take Kuvan.

[Storage] Storage temperature is lower than 25 °C.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Infant severe myoclonic epilepsy (Dravet syndrome)

Dioxenol

【Product name】DIACOMIT

[English common name]Stiripentol

[Chinese common name]Dioxenol

[English name]

[Chinese other name]

[indications]Severe myoclonic epilepsy in infants(Dravet syndrome),Dioxenol is effective in reducing the frequency of seizures in children.

[Usage and Dosage] The recommended oral dose of DIACOMIT is 50 mg / kg / day, divided into 2 or 3 divided doses (ie 16.67 mg / kg three times daily or 25 mg / kg twice daily). If the exact dose cannot be reached at the available intensity, it is as close as possible to the nearest dose, usually in the range of 50 mg to 150 mg of the recommended 50 mg / kg / day. This dose can be achieved using a combination of two DIACOMIT intensities. The maximum recommended total dose is 3,000 mg/day.

[Storage] Store at room temperature between 68 °F and 77 °F (20 °C to 25 °C).

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Mitochondrial myopathy with hyperlactosis and stroke-like episodes (MELAS syndrome)

Sodium dichloroacetate

【Product name】Sodium Dichloroacetate

[English common name]Sodium dichloroacetate

[Chinese common name]Sodium dichloroacetate

[English name]Acetic acid, 2,2-dichloro-, sodium salt

[Chinese other name]Sodium 2,2-dichloroacetate

[indications]

【Dosage】

[Storage]

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Osteogenesis imperfecta (brittle bone disease)

Bisphosphonate

【Product name】Bisphosphonates

[English common name]Bisphosphonates

[Chinese common name]Bisphosphonate

[English name]

[Chinese other name]

[indications]
Stannous methylene diphosphonate for injection: This product is only used to prepare 锝[99 Tcm ] methylene diphosphonate injection.
锝[99Tc]methylene diphosphonate injection:Rheumatoid arthritisSuch as autoimmune diseases and orthopedic diseases.

【Dosage】
Stannous methylene diphosphonate for injection: Take 1 bottle (branch) of this product, add 1-5ml of sorghum [99Tcm] sodium injection into the bottle under aseptic conditions, shake it thoroughly, so that The lyophilizate was dissolved and allowed to stand at room temperature for 20 minutes to prepare a mash [99Tcm] methylene diphosphonate injection.
锝[99Tc]methylenebisphosphonate injection: Before use, inject 5ml of agent A into the B bottle under aseptic conditions, shake well, dissolve the lyophilizate, and let stand at room temperature. In minutes, 锝[99Tc]methylenebisphosphonate injection was prepared. Intravenous injection, once a day, 20 days for a course of treatment. According to the needs of the disease, you can increase the dose and extend the course of treatment, or follow the doctor's advice.

[Storage]
Stannous methylene diphosphonate for injection: sealed, usually stored in the dark at 2-8 ° C (it is recommended to store under freezing conditions below 0 ° C).
锝[99Tc]methylene diphosphonate injection: sealed and stored in the dark at 2-8 °C.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Reiter syndrome

Serotonin

【Product name】

[English common name]

[Chinese common name]

[English name]

[Chinese other name]

[indications]

【Dosage】

[Storage]

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Congenital factor XIII deficiency , hemophilia

Human coagulation factor XIII

【Product name】Corifact

[English common name]Factor XIII concentrate

[Chinese common name]Human coagulation factor XIII concentrate

[English name]

[Chinese other name]

[indications]Congenital factor XIII deficiencyThe patient's hemorrhage.

[Usage and dosage] is only for intravenous use. Formulated before use.
Initial dose
(1) 40 international units (units) per kilogram of body weight
(2) Dosage rate should not exceed 4 mL per minute
Subsequently administered
(1) The application of the recent glutathione FXIII activity level was used to guide administration, and the dosing level was maintained at 5% to 20% every 28 days (4 weeks). It is recommended that the FXIII activity level be <5% or >20%, and the patient's clinical condition should be adjusted by ±5 units per kg of drug administration.
(2) Dosing adjustments should be guided according to the specific analysis used to determine FXIII levels. Table 1 below is an overview of an example of a dose adjustment using Berichrom® activity analysis.
Preparation and preparation
(1) The storage vial is in the original box and in the refrigerator at 2-8 ° C (36-46 ° F). Do not freeze.
(2) Prepare Corifact with USG 20 mL sterile water for injection before use.
Administration
(1) Do not mix with other drugs. Administered by separate infusion lines.

[Storage]
(1) Store the Corifact in the refrigerator at 2 – 8 ° C (36 – 46 ° F). Corifact is stable for 24 months until the expiration date on the box and vial label. Within the deadline, Corifact can be stored at room temperature not exceeding 25 ° C (77 ° F) for up to 6 months.
(2) Do not put it back in the refrigerator when the product is stored at room temperature. Clearly mark the date of storage at room temperature on the label of the box.
(3) Do not use more than the expiration date on the box and vial label, or r the end of the room storage period to see which one expires first.
(4) In order to protect from light, store the vial in the original box. Do not freeze.
(5) Immediately after administration. This product does not contain preservatives. This product must be used within 4 hours of preparation. Do not put the prepared solution in the refrigerator or freeze it.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Severe combined immunodeficiency

Human gamma globulin

【Product name】Human γ-globulin

[English common name]Human Normal Immunoglobulin

[Chinese common name]Human gamma globulin

[English name]

[Chinese other name] Γ-human globulin

[indications]
1. Mainly used to prevent measles or relieve symptoms.
2. For the prevention and treatment of viral infections such as infectious hepatitis, measles, chickenpox, mumps and herpes zoster.
3. forasthmaEndogenous allergic diseases such as allergic rhinitis and eczema.
4. Used to improve the body's immune function:
(1) Primary immunoglobulin deficiency, common variant immunodeficiency disease, immunoglobulin G subtype deficiency disease, etc.
(2) secondary immunoglobulin deficiency diseases, such as severe infection, neonatal sepsis.
(3) autoimmune diseases such as primaryThrombocytopeniaAster, Kawasaki disease.

【Dosage】
Primary thrombocytopenic purpura: 400mg/kg daily for 5 consecutive days, maintenance dose: 200 ~ 400mg / kg each time, the interval of administration depends on the condition, usually once a week. Severe infection: 200 ~ 300mg / kg per day, for 2 to 3 days. Usage: intravenous drip or diluted 5% glucose injection 1 to 2 times after intravenous infusion, the initial drip rate is 1.0ml / min, no adverse reactions after 15 minutes can speed up the drip rate, the fastest can not exceed 3.0ml / minute.

[Storage]seal.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Crohn's disease

Tacros

【Product name】Putti, Envarsus, MODIGRAF, PROGRAF, Tacrolimus, ASTAGRAF

[English common name]Tacrolimus

[Chinese common name]Tacros

[English name]Big ring piperon, Taikelima

[Chinese other name]Macrocyclic melam, texley horse

[indications]
Tacrolimus Sustained Release Capsule: Prevents graft rejection after liver or kidney transplantation.
Tacrolimus injection:
1. Prevention of graft rejection after liver or kidney transplantation.
2. Treatment of graft rejection after liver or kidney transplantation using other immunosuppressive drugs.
Tacrolimus ointment: This product is suitable for patients with moderate to severe atopic dermatitis who are not suitable for traditional treatment due to potential risks, or who have not responded to traditional therapies or cannot tolerate traditional therapy, as short-term or intermittent long-term treatment. .
Tacrolimus capsule: Prevents graft rejection after liver or kidney transplantation.

【Dosage】
Tacrolimus Sustained Release Capsules:
1. Recommended starting dose for oral administration of Prograf in adults after surgery:
(1) For patients with liver transplantation, the initial oral dose should be 0.1-0.2 mg/kg per day by weight, taken orally twice, and started 6 hours after surgery.
(2) For kidney transplant patients, the initial oral dose should be 0.15-0.3 mg/kg per day by weight, divided into two orally, and the drug should be started within 24 hours after surgery.
2. Rejection that is ineffective against traditional immunosuppressive therapy:
(1) For patients with rejection and traditional immunosuppressive therapy, patients should start treatment with Prograf. The recommended starting dose is the same as the level of first immunosuppressive agent.
Tacrolimus injection: The following recommended doses for oral and intravenous administration are only general indicators. The actual dose of the drug should be adjusted according to the needs of individual patients. The recommended dose is only the initial dose, so the treatment should be The clinical judgment was supplemented with monitoring of the concentration of tacrolimus in the blood to adjust the dose.
1. Oral administration: The daily dose is administered in two divided doses. It is best to take capsules on an empty stomach or at least 1 hour before eating or 2-3 hours after eating to achieve maximum absorption. Oral capsules are usually administered continuously to inhibit transplant rejection and have no limitations during treatment.
2. Intravenous administration:
(1) The concentrate for infusion must be diluted with 5% glucose or physiological saline in polyethylene or glass bottles.
Tacrolimus ointment:
1. Adult 0.03% and 0.1% tacrolimus ointment Apply a thin layer of this product on the affected area, gently rub it, and cover it completely, twice a day, until the atopic dermatitis symptoms and signs disappear one week. Encapsulation therapy may promote systemic absorption and its safety has not been evaluated. This product should not be used for external application of package dressings.
2. Children 0.03% tacrolimus ointment Apply a thin layer of this product on the affected area, gently rub it, and cover it completely, twice a day, until a week after the disappearance of atopic dermatitis symptoms and signs. Encapsulation therapy may promote systemic absorption and its safety has not been evaluated. This product should not be used for external application of package dressings.
Tacrolimus capsule:
1. Recommended starting dose for oral administration of Prograf in adults after surgery:
(1) For patients with liver transplantation, the initial oral dose should be 0.1-0.2 mg/kg per day by weight, taken orally twice, and started 6 hours after surgery.
(2) For kidney transplant patients, the initial oral dose should be 0.15-0.3 mg/kg per day by weight, divided into two orally, and the drug should be started within 24 hours after surgery.
2. Rejection that is ineffective against traditional immunosuppressive therapy:
(1) For patients with rejection and traditional immunosuppressive therapy, patients should start treatment with Prograf. The recommended starting dose is the same as the level of first immunosuppressive agent.
(2) The patient is converted from cyclosporine to prograf, and the first dose interval of Prograf is no more than 24 hours.

[Storage]
Tacrolimus Sustained Release Capsules: sealed, stored in a cool, dry place.
Tacrolimus injection: shading, sealed and preserved.
Tacros ointment: sealed.
Tacrolimus capsule: sealed, placed in a cool place.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
X interlocking ichthyosis

Tazarotene
other

Oral live vaccine for type 4 and type 7 adenovirus

【Product name】Ardovax

[English common name]Adenovirus types 4 and 7 live

[Chinese common name]Oral live vaccine for type 4 and type 7 adenovirus

[English name]

[Chinese other name]

[indications]

【Dosage】

[Storage]

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Music is

[English common name] Tazarotene

[Chinese common name] Tazarotene

[English name]

[Chinese other name]

[Indications]
Tazarotene gel: This product is suitable for the treatment of
Glutamine

Yiyi Maibu

【Product name】Zetia

[English common name]Ezetimibe

[Chinese common name]Yiyi Maibu

[English name]ZETIA, Ezetimibe

[Chinese other name]Etimibe, ezetimibe

[indications]
(1) Hypercholesterolemia After the conventional dose of statin treatment, the cholesterol level is still not up to standard;
(2) Unsuitable or intolerant of statins in the treatment of patients with hypercholesterolemia;
(3) patients with severe hypercholesterolemia can be directly combined with ezetimibe and small and medium dose statins;
(4)Homozygous familial hypercholesterolemiapatient;
(5) combined with fenofibrate for mixed dyslipidemia with triglyceride elevation as the main manifestation;
(6) in combination with small and medium dose statins for patients with chronic kidney disease;
(7) Application of ezetimibe alone or in combination with statins for primary or secondary prevention of coronary heart disease.

【Dosage】Patients should adhere to a proper low-fat diet during the treatment of this product. The recommended dose of this product is once a day, 10mg each time, can be taken alone or in combination with statins. This product can be taken at any time within 1 day, can be taken on an empty stomach or at the same time as food. Older patients do not need to adjust the dose. Children and adolescents over the age of 10 do not need to adjust the dose. This product is not recommended for children younger than 10 years old. Patients with mild hepatic impairment do not need to adjust the dose (Child-Pugh score at 5 or 6) (see pharmacokinetics). Patients with impaired renal function do not need to adjust the dose. Use with a bile acid sequestrant: Take this product more than 2 hours before taking the bile acid chelating agent or more than 4 hours after taking it.

[Storage] shading, sealed storage (30°C)

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
scleroderma

Iloprost

【Product name】CellCep

[English common name]Mycophenolate mofetil

[Chinese common name]Mycophenolate mofetil

[English name]Mycophenolate mofetil

[Chinese other name]Mycophenolate mofetil

[Indications] treatment of moderate primarypulmonary hypertension.

[Usage and Dosage] Adult: Each inhalation should start from 2.5 micrograms (the dose provided by the mouthpiece in the inhalation device). The dose of iloprost can be gradually increased to 5.0 micrograms depending on the needs and tolerance of different patients.
According to the needs and tolerance of different patients, iloprost should be inhaled 6-9 times a day.
Depending on the dose of drug required for the mouthpiece and nebulizer, each inhalation time should be approximately 5-10 minutes.
Patients with renal function or hepatic insufficiency: patients with abnormal liver function and renal failure requiring hemodialysis, the clearance rate of iloprost is reduced, and the dosage should be considered.
Children and adolescents (under 18 years of age): There is currently no experience in children or adolescents. This medicine cannot be used in patients under the age of 18 unless it is supported by the data.
Use of nebulizer: If a nebulizer meets the following criteria, it is considered to be suitable for atomization of the drug solution: the median aerodynamic diameter (MMAD) or median diameter (MMD) of the droplet is 3 -4m; oral output dose: 2.5 or 5 ug per inhalation of iloprost; atomization time of 2.5 or 5 ug of iloprost: approximately 4-10 minutes (to avoid systemic side effects, 4 The iloprost output in minutes must not exceed 5ug).

[Storage]Shading and confined storage.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.
Gaucher disease

Imiglucerase

【Product name】Cerezyme

[English common name]Imiglucerase

[Chinese common name]Imiglucerase

[English name]Cerezyme

[Chinese other name]Brain enzyme

[indications]Gaucher diseaseSymptom improvement (anemia,Thrombocytopenia, hepatosplenomegaly and skeletal symptoms).

【Dosage】As an immunoglucanase (genetic recombination), 60 units/kg body weight is infused every other week for 1 to 2 hours, or an appropriate dose is administered at an infusion rate of no more than 1 unit/kg/min.
For dosing, 1 vial was dissolved in 10.2 mL of water for injection and 10.0 mL was collected per vial. The desired amount of chemical solution is diluted with physiological saline to a final volume of 100-200 ml.
Incidentally, it should be increased or decreased depending on the degree of symptoms.
Further, after a period of administration, the therapeutic effect is judged, and when a sustained improvement state is observed, the maintenance dose can be reduced from the initial amount. The treatment effect can be carefully observed and the body weight can be further reduced at intervals of 3 to 6 months.

[Storage]Store at 2-8 °C in the dark.

[Tips] Some of the information on this site comes from the Internet and is only used internally by pharmacists or medical staff. It is not a substitute for doctors to diagnose face to face. Please consult a professional pharmacist for specific medications. Please refer to the actual product manual or the actual product for the product content.

Congenital factor XIII deficiency , hemophilia