RECORDATI RARE DISEASES SARL

Carbaglu helps to eliminate excessive levels of ammonia in the blood, which can be toxic to the brain.

Dosage form: tablets, dispersible

ATC: A16AA05

This medicine is used for the treatment of certain types of cancer, including Wilms tumor (a kidney tumor that usually affects children), rhabdomyosarcoma (a rare muscle tumor), testicular cancer (non-seminomatous metastatic), and other neoplasms such as melanoma or neuroblastoma.

Dosage form: powder for solution for injection

ATC: L01DA01

It is used as an add-on treatment for homocystinuria, a genetic disorder that causes the accumulation of homocysteine in the body. The medicine helps to reduce the high levels of this substance.

Dosage form: powder, oral

ATC: A16AA06

It is used to reduce the amount of cystine crystals on the surface of the eye (cornea) in adults and children from 6 months of age suffering from cystinosis.

Dosage form: solution, eye

ATC: S01XA21

It is prescribed for the treatment of cystinosis, a rare hereditary disease that causes the accumulation of an amino acid called cystine in various organs, such as the kidneys, eyes, muscles, pancreas, and brain.

Dosage form: capsules, hard gelatin

ATC: A16AA04

It is used to treat the breakdown of red blood cells (hemolytic anemia) in adults with cold agglutinin disease (CAD). This helps to reduce anemia and decrease fatigue.

Dosage form: solution for infusion

ATC: L04AJ04

It is used in adults to treat endogenous Cushing's syndrome, a condition characterized by the excessive production of cortisol by the body.

Dosage form: tablets, film-coated

ATC: H02CA02

It is used to treat maple syrup urine disease (MSUD) in patients from birth, particularly when a sudden worsening of the disease occurs and it is not possible to follow a special diet free of certain amino acids.

Dosage form: solution for infusion

ATC: B05BA01

It is used to treat acute attacks in patients suffering from acute hepatic porphyria, a disease that causes the accumulation of certain substances in the liver and symptoms such as abdominal pain, nausea, vomiting, and constipation.

Dosage form: solution for infusion, concentrate

ATC: B06AB01

It is used to facilitate the closure of the ductus arteriosus (a blood vessel near the heart) that has not closed correctly at birth, a condition called patent ductus arteriosus.

Dosage form: solution for injection

ATC: C01EB16

Signifor is used for the treatment of acromegaly and Cushing's disease in adult patients (for Cushing's disease, when surgery is not indicated or has not been effective).

Dosage form: solution for injection

ATC: H01CB05

It is used to treat vitamin E deficiency caused by digestive malabsorption in patients from birth up to 18 years of age suffering from chronic cholestasis.

Dosage form: solution, oral

ATC: A11HA08

It is used for the treatment of Wilson's disease, a rare hereditary disorder that prevents the body from properly eliminating copper, causing it to accumulate in organs such as the liver, eyes, and brain.

Dosage form: capsules, hard gelatin

ATC: A16AX05