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 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 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 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 …
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 …
Our synthetic neurotrophin mimetics may be used for treatment of diseases in which a defective trophic condition is involved in the pathogenesis, such as very important ilnesses of the Central Nervous System, like Amyotrophic Lateral Sclerosis and Parkinson’s Disease.
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
Recordati today announced that the FDA has granted Orphan Drug Designation to Recordati Rare Disease’s investigational product REC 0559 for the treatment of neurotrophic keratitis. REC 0559 is a low molecular weight non-peptidic human nerve growth factor (NGF) mimetic currently under global development by Recordati. REC 0559 was licensed in …
Recordati announced the signing of an exclusive license agreement with MimeTech for the development and subsequent commercialization on a global basis of a low molecular weight peptidomimetic of human nerve growth factor (NGF) for the treatment of neurotrophic keratitis, which already received an Orphan Drug Designation in the EU. Additional …
On 16/12/2014, orphan designation (EU/3/14/1400) was granted by the European Commission to MIMETECH S.r.l., Italy, for (1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine for the treatment of neurotrophic keratitis. Patients with neurotrophic keratitis have lower than normal levels of growth factors that are normally supplied by the trigeminal nerve and which play an important role …
