Help4Rare

List of diseases

The current experience of Help 4 Rare experts includes more than 70 diseases in which treatment have been initiated successfully or a different form of therapy has been indicated.


a b c d e f g h i j k l m n o p r s t u w x z
  • Acute biphenotypic leukaemia
  • Acute lymphoblastic leukemia
  • Acute myeloid leukemia
  • Adrenoleukodystrophy
  • Amyotrophic lateral sclerosis
  • Ataxia-telangiectasia
  • Atypical Autism
  • Blindness and visual impairment
  • Cerebral palsy
  • Chondromalacia
  • Chronic granulomatous disease
  • Chronic lymphocytic leukemia
  • Chronic myeloid leukemia
  • Common variable immunodeficiency
  • Congenital malformation syndromes
  • DiGeorge Syndrome
  • Disorders of Optic Nerve (n. II) and Visual Pathways
  • Dystonia
  • Early-onset cerebellar ataxia
  • Encephalopathy
  • Essential thrombocythemia
  • Ewing's sarcoma
  • Fanconi anemia
  • Fatty acids metabolism disorders
  • Flaccid Paralysis
  • Friedreich's ataxia (autosomal, recessive)
  • Graft versus Host disease
  • Hemiparesis
  • Hereditary retinal degeneration
  • Hereditary spinal muscular atrophy
  • Histiocytosis
  • Hodgkin’s lymphoma
  • Hypoxic brain injury
  • Idiopathic Peripheral Neuropathy
  • Ischemic stroke
  • Juvenile myelomonocytic leukemia
  • Knee Osteoarthritis
  • Late-onset cerebellar ataxia
  • Metabolic disorders
  • Miopathy
  • Multiple myeloma
  • Multiple sclerosis
  • Muscular dystrophy
  • Neuroblastoma
  • Neurodegenerative Diseases
  • Neuromuscular Disorders
  • Neuronal Ceroid-Lipofuscinoses
  • Non-Hodgkin lymphoma
  • Optic neuritis
  • Paraplegia
  • Paroxysmal nocturnal hemoglobinuria
  • Pervasive developmental disorder
  • Plasma cell leukemia
  • Polycythemia vera
  • Polyneuropathy
  • Prolymphocytic leukemia
  • Refractory Anemia with Excess Blasts (RAEB)
  • Refractory Anemia with Excess Blasts in Transformation(RAEB-T)
  • Severe aplastic anemia
  • Severe combined immunodeficiency
  • Severe perinatal asphyxia
  • Spastic paraplegia
  • Spastic quadriplegia
  • Sphingolipidosis
  • Spina bifida with hydrocephalus
  • Spinal cord injury
  • Spinal Cord Injury Cervical
  • Spinal Cord Injury Lumbar
  • Spinal Cord Injury Thoracic
  • Spinocerebellar ataxia
  • Traumatic brain injury
  • Waldenström's macroglobulinemia
  • Wiskott-Aldrich syndrome
  • X-linked lymphoproliferative disease