Sjögren’s Syndrome

Sjögren’s Syndrome Synonym(s) Sicca syndrome ORPHA378 ICD-10 H35.0 UMLS C1527336 MedDRA 10040767 Status Under evaluation What is Sjögren’s syndrome Sjögren’s syndrome is a chronic autoimmune disorder characterized by the triad of dry eyes (keratoconjunctiva sicca), sensation of oral dryness (xerostomia) and/or swelling of the salivary glands secondary to lymphocytic infiltration and destruction of the exocrine …

Sickle cell disease

Sickle Cell Disease Synonym(s) Sickle Cell Anemia ORPHA232 ICD-10 D57.0 D57.1 D57.2 UMLS C0002895 MedDRA 10040641 Status Under evaluation What is sickle cell disease? Sickle cell disease is a genetic disease in which the red blood cells become rigid and sticky, and change from being disc-shaped to being crescent-shaped (like a sickle). The change in …

Retinitis pigmentosa

Retinitis Pigmentosa ORPHA791 ICD-10 H35.5 UMLS C0035334 MedDRA 10038914 Status Under evaluation What is retinitis pigmentosa Retinitis pigmentosa is a group of hereditary diseases of the eye that lead to progressive loss of sight. In patients with retinitis pigmentosa, cells in the retina (the light-sensitive surface at the back of the eye) become damaged …

Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis Synonym(s) Charcot disease, Lou Gehrig disease Synonym(s) Charcot disease, Lou Gehrig disease ORPHA378 ICD-10 G12.2 UMLS C1527336 MedDRA 10040767 Status Under evaluation What is Amyotrophic Lateral Sclerosis? Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by progressive muscular paralysis reflecting degeneration of motor neurons in the primary motor cortex, corticospinal tracts, brainstem …

Ophthalmic diseases

Our synthetic neurotrophin mimetics are intended for treatment of corneal lesions, either spontaneous – the neurotrophic keratitis – or traumatic, or even iatrogenic, as is the case for laser treatment of miopy. In this connection, on 16/12/2014, our lead compound was designated as orphan drug for the treatment of neurotrophic …

Dermatologic diseases

Pressure ulcers, also known as pressure sores, bedsores, and decubitus ulcers, are localized injuries to the skin and/or underlying tissue that usually occur over a bony prominence, as a result of pressure, or pressure in combination with shear and/or friction. The most common sites are the skin overlying the sacrum, coccyx, heels or the …

Solid organ transplant

Solid organs destined to transplantation undergo ischemic damage as soon as they are explanted and then during their storage period. An additional injure, due to reperfusion, supervenes at the moment of their re-implant in the recipient. This medical field is, therefore, one of the most representative conditions of the Ischemia/Reperfusion (I/R) damage, …

Rare diseases

Rare diseases Rare diseases under evaluation Amyotrophic lateral sclerosis Synonym(s) Charcot disease, Lou Gehrig disease ORPHA378 ICD-10 G12.2 UMLS C1527336 MedDRA 10040767 Status Under evaluation Retinitis Pigmentosa ORPHA791 ICD-10 H35.5 UMLS C0035334  MedDRA 10038914 Status Under evaluation Sickle Cell Disease Synonym(s) Sickle Cell Anemia ORPHA232 ICD-10 D57.0 D57.1 D57.2 UMLS C0002895 MedDRA 10040641 Status Under evaluation Sjögren’s syndrome Synonym(s) Sicca syndrome ORPHA803 ICD-10 H35.0 UMLS C0002736 MedDRA 10002026 …