# OrphaNews Europe : 27 August 2014 (TEXT) **URL:** \ **Category:** General\ **Created:** [August 27, 2014, 7:48pm UTC](https://forum.livingwithataxia.org/t/orphanews-europe-27-august-2014-text/2356 "2014-08-27T19:48:08Z")\ **Posts on this page:** 1\ **Page:** 1
**Author:** ![Alan\_T](https://yyz1.discourse-cdn.com/flex027/user_avatar/forum.livingwithataxia.org/alan_t/32/31_2.png) [@Alan\_T](https://forum.livingwithataxia.org/u/Alan_T)\ **Post date:** [August 27, 2014, 7:48pm UTC](https://forum.livingwithataxia.org/t/orphanews-europe-27-august-2014-text/2356/1 "2014-08-27T19:48:08Z")
| | | | | | | | **Editorial** | | | [Ireland and Scotland adopt national rare disease plans and Spain updates its national plan](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44357) | | * * * | | | | | **EU Policy News** | | | | | | [ERRATUM: article on “First Conference on European Reference Networks sheds light on the future of ERNs” published 17 July 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44358) | | | [Recommendations on improving haemophilia patient management in Italy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44360) | | | [Haemophilia centre accreditation in Italy could serve as a model for other European countries and other rare diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44362) | | | [European Guidelines for the Certification of Haemophilia Centres](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44363) | | * * * | | | | | **National & International Policy Developments** | | | | | | [National strategies to improve paediatric cancer treatment in low- and middle-income countries](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44365) | | | | | **Other International News** | | | [Patient-initiated guidance on clinical development of treatment for Duchenne Muscular Dystrophy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44366) | | | [NIH partners with American medical universities to accelerate diagnosis of rare diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44367) | | | [A new research organisation dedicated to Down syndrome: Trisomy 21 Research Society (T21RS)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44368) | | | | | **Guidance Documents and Recommendations** | | | [Congenital muscular dystrophy: review of diagnostic approaches made by the International Committee on the Standard of Care for Congenital Muscular Dystrophies](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44465) | | | [Ocular myastheny: European Federation of Neurological Societies and European Neurological Society guidelines for the treatment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44466) | | | [Hereditary non-polyposis colon cancer: guidelines on genetic evaluation and management from the US Multi-Society Task Force on Colorectal Cancer](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44467) | | | [Idiopathic pulmonary fibrosis: French practical guidelines on diagnosis and management](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44468) | | | [Maple syrup urine disease: nutrition management guideline](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44469) | | | | | **Bioinformatics, Registries and Data Management** | | | | | | [The need to build minimum and common data sets to research, diagnose and treat rare diseases more efficiently](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44370) | | | [Internet tools as effective methods to recruit rare disease patients into registries](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44371) | | | [The long road to constituting the Italian national rare disease registry](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44372) | | | | | **Screening and Testing** | | | | | | [Next generation sequencing is more rapid and cost effective to diagnose hereditary Alport syndrome](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44374) | | | [Advances in genetic screening tools throw light on genetic causes of epilepsy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44375) | | * * * | | | | | **New Syndromes** | | | | | | [Novel form of myopathy linked to a homozygous mutation in TOR1AIP1 in a consanguineous family](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44385) | | | [Novel neurodegenerative phenotype associated to a homozygous PCNA missense mutation in a family](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44386) | | | [Tall stature, severe lateral tibial deviation, scoliosis, hearing impairment, camptodactyly, and arachnodactyly due to a homozygous mutation in FGFR3 in two brothers](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44387) | | | [A mitochondrial origin for frontotemporal dementia and amyotrophic lateral sclerosis through CHCHD10 involvement in a large family](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44388) | | | [Prenatal growth restriction, retinal dystrophy, diabetes insipidus and white matter disease associated with a missense mutation in PRPS1 in two male siblings](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44389) | | | [Congenital myopathy with fatigable weakness responding to pyridostigimine linked to mutations in RYR1 in two siblings](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44390) | | | [A novel overgrowth condition including macrocephaly, obesity, speech delay and advanced carpal ossification is due to heterozygous mutations in SETD2 in two patients](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44391) | | * * * | | | | | **New Genes** | | | | | | [Autosomal recessive epileptic encephalopathy with seizure onset in the first day of life is caused by heterozygous mutations in SLC13A5 in two families](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44393) | | | [Autosomal recessive cone rod dystrophy due to mutations in POC1B](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44394) | | | [Ectopic progenitors and neuronal heterotopia in mouse and human are linked to mutations in EML1](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44395) | | | [Limb-girdle muscular dystrophy type 1G caused by mutations in HNRPDL in two families](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44396) | | | [Recessive non-syndromic, prelingual, profound hearing loss due to a splice site mutation in FAM65B in a consanguineous Turkish kindred](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44397) | | | [Blackfan-Diamond anemia caused by RPS29 mutations in two families](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44398) | | | [Blackfan-Diamond anemia with mandibulofacial dystostosis associated with TSR2 and RPS28 mutations](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44399) | | | [46, XY partial gonadal dysgenesis linked to increased gene copy numbers of VAMP7](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44400) | | | [Behr syndrome is due to homozygous nonsense mutation in C12orf65 in four patients from three unrelated families](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44401) | | | [Impaired neuromuscular transmission is associated with mutations of the SLC25A1 gene](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44402) | | | [Intracranial germ cell tumours linked to JMJD1C germline mutations and to KIT/RAS signaling pathway somatic mutations](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44403) | | | [X-linked recessive intellectual disability with features of 3C syndrome associated with missense variant in CCDC22 in an Austrian family with two affected male children](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44404) | | | [Microphthalmia and anterior segment dysgenesis caused by novel mutations in PXDN](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44405) | | | [Activating mutations in RRAS underlie a phenotype within the RASopathy spectrum and contribute to leukaemogenesis](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44406) | | | [Noonan-like syndrome with a variable phenotype ranging from severe to very mild is associated with heterozygous germline mutations in A2ML1](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44407) | | | [Mitochondrial encephalomyopathies due to VARS2 and TARS2 mutations](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44408) | | | [Genetic variants of the KCNQ1 gene confer a risk of Beckwith–Wiedemann syndrome upon maternal transmission](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44409) | | | [Leri pleonosteosis results from microduplication at 8q22.1 encompassing GDF6 and SDC2 in two families](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44410) | | * * * | | | | | **Research in Action** | | | | | | | | **Clinical Research** | | | [Infantile neuronal ceroid lipofuscinosis: combination therapy with cysteamine bitartrate and N-acetylcysteine is beneficial for the patients](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44412) | | | [Sickle cell anemia: non-myeloablative allogeneic hematopoietic stem cell transplantation allows for complete replacement with circulating donor red blood cells](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44413) | | | [Hepatocellular carcinoma: everolimus did not improve overall survival in patients whose disease progressed during or after receiving sorafenib](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44414) | | | [B-cell chronic lymphocytic leukemia: ibrutinib significantly improves progression-free survival and response rate compared to ofatumumab](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44415) | | | [Metastatic enteropancreatic neuroendocrine tumors: lanreotide significantly prolonged progression-free survival](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44416) | | | [Pediatric autoimmune and inflammatory central nervous system diseases: rituximab should be restricted to disorders with significant mortality due to its infectious risk](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44417) | | | [Acquired thrombotic thrombocytopenic purpura: preemptive rituximab infusions after remissions efficiently prevent relapses](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44418) | | | [Phenylketonuria: rAvPAL-PEG is safe and reduces blood phenylalaline concentrations even though it develops antibodies against polyethylene glycol](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44419) | | | [Hutchinson-Gilford progeria syndrome: increased survival with protein farnesylation inhibitors](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44420) | | | [Shared medical appointments improve quality of life in neuromuscular patients](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44421) | | | [Thrombotic thrombocytopenic purpura: active monitoring and management during pregnancy results in positive pregnancy outcomes](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44422) | | | [Malaria: the early concentration of once-infected erythrocytes is a solid candidate marker to predict post-artesunate delayed hemolysis](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44423) | | | [Somatic NLRP3 mosaicism underlying Muckle-Wells syndrome](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44424) | | | [Ornithine transcarbamylase deficiency: clues for early diagnosis and timely treatment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44425) | | | | | **Stem Cells** | | | | | | [Amyotrophic lateral sclerosis: focal transplantation of human iPSC-derived glial-rich neural progenitors improves lifespan of mice](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44434) | | | | | **Gene Therapy** | | | [Spontaneous autoimmune peripheral polyneuropathy: vasoactive intestinal polypeptide expressing dendritic cells protects against the disease](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44435) | | | [Leber congenital amaurosis: CEP290 gene transfer rescues the cellular phenotype](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44436) | | | | | **Therapeutic Approaches** | | | | | | [Rett syndrome: functional recovery and phenotype amelioration with recombinant human IGF1 and β2-adrenergic receptor agonist treatments in a mouse model](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44427) | | | [Duchenne muscular dystrophy: effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in mdx mice](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44428) | | | [Severe spinal muscular atrophy: human growth hormone increases survival motor neuron expression and survival in mouse model](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44429) | | | [Amyotrophic lateral sclerosis: treatment with an antibody directed against Nogo-A delays disease progression in mouse model](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44430) | | | [Huntington disease: Xpro1595, a dominant negative inhibitor of soluble TNF-α, suppresses inflammatory responses and improves the impaired motor function in model mice](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44431) | | | [Head and neck cancer: on-demand intracellular amplification of chemoradiation with cancer-specific plasmonic nanobubbles improves efficacy of chemoriadiation](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44432) | | | | | **Diagnostic Approaches** | | | | | | [Arthrogryposis syndrome: diagnostic approach to etiology, classification, genetics, and general principles](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44438) | | | [‘Clinical Face Phenotype Space’, an algorithm extracting phenotypic information from photographs, is relevant for diagnosis in eight syndromes with dysmorphic features](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44439) | | | [Neuro-cardio-facio-cutaneous syndromes: combined MPS-Sanger sequencing based strategy as an effective diagnostic tool for heterogeneous diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44440) | | | [Improving molecular diagnosis in epilepsy by a dedicated high-throughput sequencing platform](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44441) | | | [Idiopathic inflammatory myopathy: combining MRI and muscle biopsy improves diagnostic accuracy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44442) | | | [Whole-body muscle MRI to detect myopathies in non-extrapyramidal bent spine syndrome](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44443) | | * * * | | | | | **Patient Management and Therapy** | | | [Congenital heart malformation: cardiovascular management in pregnancy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44444) | | | [Goldenhar syndrome: early ultrasound screening should be performed in all infants born with the disorder because of a high rate of associated anomalies](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44445) | | | [Classic galactosemia: a re-evaluation of life-long severe galactose restriction for the nutrition management](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44446) | | | [Gastric cancer: review on angiogenesis inhibitors](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44447) | | | [Anaplastic oligodendroglioma and oligoastrocytoma: review on adjuvant treatment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44448) | | | [Coenzyme Q10 deficiency syndrome: four articles on clinical presentation, biochemical diagnosis, genetics and therapy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44449) | | | [Uveitis associated with juvenile idiopathic arthritis: review of the treatment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44450) | | | [Anti-neutrophil cytoplasmic antibody-associated vasculitis: review of the treatment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44451) | | | [Disorder of sex development: review on transition of care for adolescents](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44452) | | | [Aggressive pituitary adenoma: review on diagnosis and emerging treatments](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44453) | | | [Cataplexy: review on clinical aspects, pathophysiology and management strategy](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44454) | | | [Neurofibromatosis type 1: review on a multidisciplinary approach to care](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44455) | | | [Giant cell arteritis and polymyalgia rheumatica: a review](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44456) | | | [Non-infectious cryoglobulinemia vasculitis: review on clinical and therapeutic approaches](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44457) | | | [Kawasaki disease: review on pathophysiology, clinical manifestations and management](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44458) | | | [Respiratory bronchiolitis - interstitial lung disease: a review](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44459) | | | [Hamartomatous polyposis syndromes: a review](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44460) | | | [IgG4-related diseases: a review](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44461) | | | [Tracheobronchopathia osteochondroplastica: a review](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44462) | | | [Eosinophilic esophagitis: a review](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44463) | | | [2 new and 8 updated GeneReviews published](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44464) | | * * * | | | | | **Orphan Drugs** | | | | | | [Need for paediatric labelling of drugs to improve treatment in children](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44377) | | | [Recommendations to improve efficiency of clinical trials for rare diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44378) | | | [A study of the literature sheds light on trends in rare disease and orphan drug research](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44379) | | | | | **Regulatory News** | | | | | | [FDA approves Ruconest to treat patients with hereditary angioedema (HAE)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44381) | | | | | | [27 positive opinions recommending orphan designation at the July 2014 COMP meeting](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44383) | | * * * | | | | | **Grants** | | | | | | [The Sturge-Weber Foundation Research Grants](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44472) | | | [Myotonic Dystrophy Foundation (MDF) Fund-a-Fellow postdoctoral grant programme](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44473) | | | [Call for tender Chafea/2014/Health/05 concerning the development of a manual and toolbox for the assessment of European Reference Networks](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44474) | | | [DEBRA International research grants](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44475) | | | [NBIA Disorders Association Announces Research Grants for Pantothenate Kinase-Associated Neurodegeneration (PKAN)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44476) | | | [FRT - Fondation René Touraine (FRT) Award](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44477) | | * * * | | | | | **Courses & Educational Initiatives** | | | | | | [EUPATI Training Course](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44479) | | | [International Summer School: Rare diseases and orphan drug registries](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44480) | | | [‘Explique-moi les essais cliniques’: The keys to understanding clinical trials to become an actor of one’s disease (in French)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44481) | | | [Genodermatoses Network Training Session](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44482) | | | [III International EPIRARE workshop: Rare disease and orphan drug registries](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44483) | | | [Quality assessment of health care guidelines for rare diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44484) | | | [Health care guidelines for rare diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44485) | | * * * | | | | | **What's on Where?** | | | | | | [Third Symposium ATP1A3 in disease: Genotype/phenotype correlations, modelling and identification of potential targets for treatment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44487) | | | [3rd Nordic Conference on Rare Diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44488) | | | [25th European Dysmorphology Meeting](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44489) | | | [National Huntington's Conference](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44490) | | | [EFGCP Multi-Stakeholder Workshop & Discussion: How to Ensure Optimal Ethical Review within the New Clinical Trials Regulation?](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44491) | | | [15th International Conference on Human Genome Variation and Complex Genome Analysis (HGV2014)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44492) | | | [International Clinical Conference Jérôme Lejeune: Intellectual disability and the importance of assessment](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44493) | | | [SIOPE – ENCCA Conference 2014: Joining Efforts for a Brighter Future for Children and Adolescents with Cancer](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44494) | | | [16th International Conference on Behçet’s Disease](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44495) | | | [2nd European Conference on Aniridia](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44496) | | | [4th Annual Brain Metastases Research and Emerging Therapy Conference](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44497) | | | [9th International Research Symposium on Marfan Syndrome and related disorders](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44498) | | | [3rd International Conference on Immune Tolerance 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44499) | | | [EFGCP / DIA / EMA Annual Conference 2014 on Better Medicines for Children: ‘Explore Ways to Enhance Collaboration Between Key Players’](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44500) | | | [Single topic symposium in metabolic liver disease](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44501) | | | [ICORD 2014 Annual Meeting: Societal value of Prevention, Diagnosis and Treatment of Rare Diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44502) | | | [The Translational Science of Rare Diseases: From Rare to Care II](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44503) | | | [9th ISNS European Neonatal Screening Regional Meeting](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44504) | | | [Dysmorphology and Radiology of Inborn Errors of Metabolism](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44505) | | | [International Scientific Symposium on Angelman Syndrome 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44506) | | | [64th Annual Meeting of the American Society of Human Genetics: ASHG 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44507) | | | [NORD’s Rare Diseases and Orphan Products Breakthrough Summit](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44508) | | | [14th International Congress on Neuronal Ceroid Lipofuscinoses (Batten Disease)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44509) | | | [International VHL Medical Symposium](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44510) | | | [30th Annual Meeting of the Histiocyte Society](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44511) | | | [ESID Meeting 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44512) | | | [New frontiers in Neuroacanthocytosis and Neurodegeneration with Brain Iron Accumulation: From Benchside to Bedside](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44513) | | | [2nd International Rare Diseases Research Consortium (IRDiRC) Conference](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44514) | | | [4th Pan-European Conference on Haemoglobulinopathies and Rare Anaemias](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44515) | | | [Third International Symposium on Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44591) | | | [Ataxia Telangiectasia Clinical Research Conference 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44516) | | | [Cilia 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44517) | | | [22nd René Touraine Foundation for Dermatology Scientific Meeting](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44518) | | | [Trisomy 21 Research Society (T21RS) International Conference](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44519) | | | [7th International Conference on Children’s Bone Health](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44520) | | | [2nd International Primary Immunodeficiencies Congress (IPIC)](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44521) | | | [13th International Congress of Human Genetics (ICHG) 2016](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44522) | | | | | | [Orphan Drugs Summit 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44524) | | | [The Orphan Drugs, Collaborations & Market Access Congress](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44525) | | | [Orphan Drugs and Rare Diseases](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44526) | | | [3rd annual World Biosimilar Congress](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44527) | | | [The World Orphan Drug Congress Europe 2014](http://www.orpha.net/actor/EuropaNews/2014/140827.html#44528) |