Diagnosis Genetic Disorders Mitochondrial Diseases Batten Disease Epidermolysis Bullosa Hemophagocytic Lymphohistiocytosis Metabolic Disorders Amyloidosis Neurological Disorders Sanfilippo Syndrome Fibrodysplasia Ossificans Progressiva Hereditary Angioedema Progressive Supranuclear Palsy Advancements in Treatment Childhood Illnesses Endomyocardial Fibrosis Clinical Trials Moebius Syndrome Orphan Diseases Mitochondrial Disease Congenital Diseases Wilkie Syndrome Multiple Sclerosis Haemophagocytic Lymphohistiocytosis Aplastic Anaemia Mitochondrial Disorders Pediatric Rare Diseases Familial Chylomicronemia Syndrome Tuberculosis Surgical Procedures Congenital Conditions Osteomalacia EPOF Healthcare Planning Multicentric Carpotarsal Osteolysis Langerhans Cell Histiocytosis Epidemiology Drug Discovery Immune Disorders Chediak-Higashi Syndrome Regulatory Framework Challenges Legal Aspects Barth Syndrome Hereditary Spastic Paraplegia Optic Atrophy Addison's Disease Cancer Fanconi Anemia Neuroblastoma Botulism Gene Therapies Nephrogenic Systemic Fibrosis Xeroderma Pigmentosum (XP) Genetic Conditions Wilson's Disease Motor Neurone Disease HLH Cushing Syndrome Kawasaki Disease Prevalence Gene Therapy Creatine Transporter Deficiency Multiple Myeloma Neurodegenerative Diseases Endocrine Disorders
Family say steroid therapy has brought faint progress in a condition that demands specialist care.
