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- ... Pombilti) to treat late onset Pompe disease (a genetic condition that causes deficiency in lysosomal acid alpha- ...
- ... much iron in their body because of a genetic blood disorder called non–transfusion-dependent thalassemia (NTDT). ...
- ... in people with tuberous sclerosis complex (TSC; a genetic condition that causes tumors to grow in many ...
- ... be used only in people with a certain genetic make-up. Your doctor may order a blood ...
- ... be used only in people with a certain genetic make-up. Your doctor may order a blood ...
- ... and walking), or tuberous sclerosis complex (TSC; a genetic condition that causes tumors to grow in many ...
- ... types of PIK3CA Related Overgrowth Spectrum (PROS; a genetic condition that causes overgrowth and abnormalities in certain ...
- ... be used only in people with a certain genetic make-up. Your doctor may order a blood ...
- ... children who have achondroplasia (ACH; achondroplastic dwarfism; a genetic condition of bone growth that results in short ...
- ... treat certain symptoms associated with alpha-mannosidosis (a genetic condition where the body is unable to break ...