GMP ORPHAN DRUGS FOR ORPHAN DISEASES

Pharmaceutical and veterinary products for the treatment of orphan diseases; medication for the treatment of orphan diseases; pharmaceutical and veterinary products for pediatric use for the treatment of orphan diseases; medication for pediatric use for the treatment of orphan diseases; chemical preparations for medical or pharmaceutical use in the treatment of childhood diseases; pharmaceutical and veterinary products and medicines for the treatment of orphan diseases, particularly spinal muscular atrophy, sickle-cell anemia, progressive nuclear palsy, amyotrophic lateral sclerosis (ALS), cystic fibrosis, Duchenne muscular dystrophy, macrophagic myofasciitis, lysosomal storage disease, leukodystrophy; pharmaceutical and veterinary products and medicines for the treatment of hyperammonemia of all kinds

[ Medical, surgical and veterinary apparatus and instruments, for diagnosis and treatment, particularly in connection with orphan diseases; medical devices containing chemical products for diagnostic use of orphan diseases; medical diagnosis apparatus and instruments to diagnose orphan diseases; automatic testing apparatus to diagnose orphan diseases, for medical use, also in the form of kits; medical apparatus and instruments in the form of kits for detecting the presence of analytes in biological fluids, other fluids and other structures; immunochemical testing apparatus for medical use; blood testing apparatus; radiological apparatus for medical use; resuscitation apparatus; medical imaging apparatus incorporating medical imaging software; needles for medical use; gloves for medical use; syringes for medical use; inhalers for medical purposes; injectors for medical use; X-ray photographs for medical use; radiology screens for medical use; receptacles for applying medicines; containers especially made for medical waste; probes for medical use; stethoscopes; heart pacemakers; thermometers for medical use; cases fitted for medical instruments for use by surgeons or doctors ]

Evaluations, assessments and research in the fields of science; research and development services in the field of medicine; research and development of new products for others, namely, medical diagnosis apparatus; research and development in the field of biotechnology; scientific research for medical purposes; scientific laboratory services in the medical field; all the aforesaid services in connection with the treatment of childhood and orphan diseases, particularly spinal muscular atrophy, sickle-cell anemia, progressive nuclear palsy, amyotrophic lateral sclerosis (ALS), cystic fibrosis, Duchenne muscular dystrophy, macrophagic myofasciitis, lysosomal storage disease, leukodystrophy

Medical services; medical and sanitary care, namely, telemedecine services including remote diagnosis or medical follow-up; medical testing for the diagnosis and treatment of persons; providing medical advice on health matters in the field of medication administration; providing medical advisory services relating to medical apparatus and instruments; medical diagnosis services of orphan diseases; providing medical information, all the aforesaid services in connection with the treatment of childhood and orphan diseases, particularly spinal muscular atrophy, sickle-cell anemia, progressive nuclear palsy, amyotrophic lateral sclerosis (ALS), cystic fibrosis, Duchenne muscular dystrophy, macrophagic myofasciitis, lysosomal storage disease, leukodystrophy