Rare disease

Showing 1–12 of 216 results

  • Crohn's disease

    5-aminosalicylic acid

    【Product name】CANASA

    [English common name]5-Aminosalicylic acid

    [Chinese common name]5-aminosalicylic acid

    [English name]5-ASA; 5-Amino salicylic acidl, Mesalazine

    [Chinese other name]5-amino-2-hydroxybenzoic acid, mesalazine

    [Indications] For the treatment of ulcerative colitis, Crohn's disease; suppositories for the treatment of ulcerative proctitis, can also be combined with mesalazine tablets.

    [Usage and Dosage] Oral: Adult, ulcerative colitis (acute attack): 4 times a day, 1g each time; maintenance treatment, 3 times a day, 0.5g each time.
    Crohn's disease: 4 times a day, 1g each time. For children over 2 years old, take 20-30mg per kilogram of body weight per day, in divided doses.
    Suppository: Adult, 1-2 times a day, 1 suppository each time. Children over the age of two, follow the doctor's advice.

    [Storage] The tablets should be stored at room temperature (15-25 °C) and should not be taken after the expiration date. Suppositories should be stored at room temperature (15-25 °C) in the original package and should not be used after the expiration date.

    [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.

  • Hyperphenylalaninemia

    5-hydroxytryptophan

    【Product name】Levothym

    [English common name]5-Hydroxytryptophan

    [Chinese common name]5-hydroxytryptophan

    [English name]5-hydroxy-D-tryptophan

    [Chinese other name]5-hydroxy-DL-tryptophan

    [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.

  • Tetrahydrobiopterin deficiency , scleroderma

    Serotonin

    【Product name】Serotonin

    [English common name]5-Hydroxytryptamine

    [Chinese common name]5-hydroxytryptamine name

    [English name]5-hydroxytryptamine

    [Chinese other name]5-hydroxytryptamine

    [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.

  • Crohn's disease

    6-巯嘌呤 (6MP)

    【Product name】MERCAPTOPURINE, PURINETHOL

    [English common name]Mercaptopurine

    [Chinese common name]6-琉嘌呤 (6MP)

    [English name]Purinethol

    [Chinese other name]

    [indications]Suitable forChorionic epithelial carcinoma,Malignant moleAcute lymphoblastic leukemia and acute non-lymphocytic leukemia, chronic granulocytic leukemia.

    【Dosage】Oral administration:
    1 Chorionic epithelial cancer: commonly used in adults, daily 6mg ~ 6.5mg / kg, divided into two oral, 10 days for a course of treatment, intermittent treatment for 3 to 4 weeks.
    2 leukemia: start, daily 2.5mg / kg or 80 ~ 100mg / m, once a day or in divided doses, generally 2 to 4 weeks after treatment can be markedly effective, such as 4 weeks after medication, no clinical improvement and The number of white blood cells is decreased, and it can be considered to be added to 5 mg/kg per day under careful observation; maintenance, 1.5 mg to 2.5 mg/kg or 50 mg to 100 mg/m per day, once a day or in divided doses.

    [Storage] shading, sealed and stored.

    [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.

  • Pompe disease

    A-glucosidase

    【Product name】Myozyme, REMINYL, RAZADYNE

    [English common name]Alglucosidase alfa

    [Chinese common name]A-glucosidase

    [English name]

    [Chinese other name]

    [Indications] LUMIZYME (alglucosidase alfa) is a lysosomal glycogen-specific enzyme for Pompe disease (acid alpha-glucosidase (GAA) deficiency) with advanced (non-infant) disease at age 8 years and older ) but there is no evidence of cardiac hypertrophy. The safety and efficacy of LUMIZYME has not been evaluated in controlled clinical trials in infant-infected patients or in advanced (non-infant) patients under 8 years of age.

    [Usage and Dosage] The recommended dose of LUMIZYME is 20 mg / kg body weight every 2 weeks for intravenous infusion.
    LUMIZYME is supplied as sterile, pyrogen free, white to white, lyophilized cake or powder for sterile water for injection, USP, to produce a concentration of 5 mg / mL; then further diluted with 0.9% sodium chloride injection USP is used for intravenous infusion. Disposable vials are only available in 50 mg doses.

    [Storage] Store LUMIZYME at a refrigerating temperature of 2° to 8°C (36° to 46°F). Do not use LUMIZYME after the expiration date on the vial. The reconstituted and diluted solution should be administered immediately. If not immediately available, the reconstituted and diluted solution can be stable for up to 24 hours at 2° to 8°C (36° to 46°F). It is not recommended to store the reconstituted solution at room temperature. The reconstituted and diluted LUMIZYME solution should be protected from light. Do not freeze or shake.

    [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.

  • Low alkaline phosphatase

    Asfotase alfa

    【Product name】Strensiq

    [English common name]Asfotase alfa

    [Chinese common name]

    [English name]

    [Chinese other name]

    [indications]For the treatment ofLow alkaline phosphatase

    [Usage and Dosage] Common dose: 2 mg / kg subcutaneous injection, 3 times a week or 1 mg / kg subcutaneous injection, 6 times a week
    Maximum dose: 9 mg / kg / week

    [Storage] Use until refrigerated conditions of 2 ° C to 8 ° C (36 ° F to 46 ° F) and protected from light.

    [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.

  • Hepatolenticular degeneration

    D-penicillamine

    【Product name】Calcium edetate injection

    [English common name]D-Penicillamine

    [Chinese common name]D-penicillamine

    [English name]3-Mercapto-D-valine

    [Chinese other name]3-mercapto-D-proline

    [Indications] for the treatment of Wilson's disease, cystineuria and severe.

    [Usage and Dosage] Wilson's Disease - The optimal dose can be determined by measuring the measurement of urinary copper excretion and free copper in serum. Urine must be collected in copper-free glassware and copper should be quantified immediately before and after treatment with CUPRIMINE.
    Determination of 24-hour urinary copper excretion during the first week of treatment with penicillamine was the most valuable. In the absence of any drug response, a dose between 0.75 and 1.5 g results in over 2 mg of copperuria for the first 24 hours should last for about 3 months, at which time the most reliable method of monitoring maintenance therapy is to determine the serum. Free copper. This is equal to the difference between the total copper and ceruloplasmin-copper quantitatively determined. Well-treated patients usually contain less than 10 mcg of free copper/dL serum. A dose of more than 2 grams per day is rarely required. If the patient is unable to receive CUPRIMINE treatment, the replacement therapy is trientine hydrochloride.
    The usual dose of CUPRIMINE for the treatment of cystineuria is 2 g/day for adults, ranging from 1 to 4 g/day. For pediatric patients, the dose can be based on 30 mg / kg / day. The total daily amount should be divided into four doses. If four equal doses are not feasible, give a larger percentage before going to bed. If the adverse reaction requires a dose reduction, it is important to maintain the bedtime dose.
    Starting doses of 250 mg/day and gradually increasing to the required amount can more closely control the effects of the drug and may help reduce the incidence of adverse reactions.

    [Storage] Keep the container closed.

    [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)

    L-arginine

    【Product name】Argi-U

    [English common name]L-Arginine

    [Chinese common name]L-arginine

    [English name](S)-2-Amino-5-guanidinopentanoic acid, L-protein amino acid

    [Chinese other name]2-amino-5-mercaptovaleric acid, L-protein amino acid

    [Indications] For the adjuvant treatment of diseases such as hepatic encephalopathy.

    [Usage and dosage] Oral. 3 to 6 tablets at a time, 3 times a day.

    [Storage] Sealed and stored in a dry 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.

  • Spinal muscular atrophy

    Nusinersen

    【Product name】SPINRAZA

    [English common name]Nusinersen

    [Chinese common name]

    [English name]

    [Chinese other name]

    [indications]Spinal muscle atrophy.

    [Usage and Dosage] Infant spinal muscular atrophy is usually administered as a dose of Nuccinellsen in the table below. They were administered 2 weeks, 4 weeks and 9 weeks after the first administration, and administered at intervals of 4 months thereafter. In either case, intrathecal administration should be carried out within 1 to 3 minutes. Spinal muscular atrophy other than infant type is usually administered once as a dose in the table below as Nuccinellsen. Administration should be carried out 4 weeks and 12 weeks after the first administration, and thereafter at intervals of 6 months. In either case, intrathecal administration should be carried out within 1 to 3 minutes.

    [Storage] Store in the refrigerator (2 ° C – 8 ° C). Do not freeze. Place the vial in an outer carton to protect it from light.
    If not refrigerated, Spinraza can be stored in its original carton, protected from light at 30 ° C or below, for up to 14 days.
    Prior to administration, if necessary, the unopened Spinraza vial can be removed and returned to the refrigerator. If removed from the original carton, the total cooling time should not exceed 30 hours at temperatures not exceeding 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.

  • Lysosomal acid lipase deficiency

    Sebelipase alfa

    【Product name】Kanuma

    [English common name]Sebelipase Alfa

    [Chinese common name]

    [English name]Se be LYE pase AL fa

    [Chinese other name]

    [indications]KANUMATM is a specific hydrolyzed lysosomal cholesterol ester and triglyceride suitable for useLysosomal acid lipase (LAL) deficiencyDiagnose the treatment of the patient.

    【Dosage】The patient presented a rapid progression of LAL deficiency in the first 6 months of life: the recommended starting dose was 1 mg/kg as an intravenous infusion once a week. For patients who did not achieve optimal clinical response, increase to 3 mg/kg once a week.
    LAL-deficient children and adult patients: The recommended dose is 1 mg/kg as an intravenous infusion once every other week.

    [Storage]Store Kanuma in the original carton at a refrigerated temperature of 2 ° C to 8 ° C (36 ° F to 46 ° F) to prevent light exposure.Do not shake or freeze the vial.

    [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.

  • Fabry disease

    Alpha-galactosidase

    【Product name】Fabrazyme

    [English common name]Agalsidase alpha

    [Chinese common name]Alpha-galactosidase

    [English name]

    [Chinese other name]

    [indications]Fabry disease.

    【Dosage】As agarase beta (genetic recombination), 1 mg / kg body weight is usually administered intravenously every other week.

    [Storage] Store at 2-8 ° C and use within 24 hours.

    [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.

  • Pompe disease

    Alpha-glucosidase

    【Product name】Myozyme

    [English common name]Alglucosidase alfa

    [Chinese common name]Alpha-glucosidase

    [English name]α-Glucosidase

    [Chinese other name]Alpha-glucosidase

    [indications]For the use of the specific lysosomal glycogenase indicated in the patient's Pompe disease (GAA deficiency).

    【Dosage】20 mg/kg by intravenous infusion every 2 weeks

    [Storage]Myozyme was stored at a refrigerated temperature of 2° to 8°C (36° to 46°F).Do not use Myozyme after the expiration date on the vial.

    [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.