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6th International Conference on Rare Diseases & Orphan Drug, will be organized around the theme “Challenges & opportunities in elimination of Rare diseases & orphan drug development ”

Rare Diseases Meet 2019 is comprised of keynote and speakers sessions on latest cutting edge research designed to offer comprehensive global discussions that address current issues in Rare Diseases Meet 2019

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Determination and the treatment of a rare disease end up confounded due to the way that numerous medicinal services providers may have confined contribution with the identification of the particular disease condition with the diagnosis of rare diseases. Also, examination analysis before manifestation onset or diagnosis early stage in the disease can be challenging. Biopharmaceutical specialists in the field have used new advances and the creating exploratory appreciation of numerous rare diseases to create and analyse ground breaking therapies in the course of the most recent 10 years. In the most recent decade, in excess of 230 new orphan therapeutic medications were recognised by the U.S. Food and Drug Administration (FDA). In 2015 alone, about half (47%) of novel most recent medication confirmations were for rare diseases. Disease specific treatments should be created for beneficial outcomes in treatment of Rare Diseases.

  • Track 1-1Congenital plasminogen deficiency
  • Track 1-2Rarity of the condition or disease
  • Track 1-3Familial chylomicronemia syndrome
  • Track 1-4Nonspecific manifestation
  • Track 1-5Clinical & phenotypic evaluation
  • Track 1-6Parechovirus B

Brain is body's control centre. It's a part of the nervous system, which likewise incorporates the spinal cord and a substantial system of nerves and neurons. Together, the nervous system controls everything from your senses to the muscles all through your body. When your brain is damaged, it can influence a wide range of things, including your memory, your sensation, and even your identity. Brain disorders incorporate any conditions or inabilities that influence your brain. Brain injuries are regularly caused by blunt trauma. Creutzfeldt-Jakob disease (CJD) is a rare, degenerative, fatal brain disease.  It influences around one individual in each one million every year worldwide in the United States there are around 350 cases per year.  CJD generally shows up in later life and runs a quick course.

 

  • Track 2-1Normal pressure hydrocephalus
  • Track 2-2Frontotemporal dementia
  • Track 2-3Transient ischemic attack
  • Track 2-4Amyotrophic lateral sclerosis
  • Track 2-5Traumatic brain injury
  • Track 2-6Creutzfeldt-jakob disease

Rare cancers can originate in any organ of the body Examples incorporate gastrinomas, childhood acute myeloid leukaemia, schwannomas, retinoblastomas, or thymomas. For a portion of these diseases, specific medications have not yet been produced. All in all, in any case, inquire about in rare cancers is greatly dynamic, with numerous medications either approved or being developed. The various kinds of rare oncological diseases incorporate tumours of the body and haematological diseases of the blood. Although independently these malignancies are uncommon, as a gathering, they represent a significant majority of individuals with cancer.  Treatment for rare cancers is typically collaboration, with general professionals, oncologists, haematologists, radiation specialists, and different physicians consulting with each other.

 

  • Track 3-1Acute leukaemia of ambiguous lineage
  • Track 3-2Chronic myeloproliferative disorders
  • Track 3-3Hyaline fibromatosis syndrome
  • Track 3-4Gastrointestinal stromal tumours
  • Track 3-5Common variable immunodeficiency

The human ordination is continually exposed to varied sources of desoxyribonucleic acid injury. Rare genetic diseases are specifically caused by changes in genes or chromosomes, often go from one generation to next. Most of these diseases are regularly connected with severe or life threatening features. Numerous rare diseases seem early in life, and around 30% of children with rare diseases will die before achieving their fifth birthday. With a single analysed patient just, ribose-5-phosphate isomerase deficiency is viewed as the rarest known genetic disease. Frequency of a single rare genetic disease can fluctuate broadly yet is generally very infrequent, usually to or less than one per 1,00,000 births. A disease is viewed as rare if it influences less than 2,00,000 individuals in the United states, or less than five for every 10,000 individuals in the European Union.

 

  • Track 4-1Marfan syndrome
  • Track 4-2Klinefelter syndrome
  • Track 4-3Albinism immunodeficiency
  • Track 4-4Haemolytic uremic syndrome

Skin disorders differ significantly in manifestations and severity. They can be brief or changeless, and might be painless or painful. Some have situational causes, while others might be hereditary. Some skin conditions are minor, and others can be dangerous. Out of these, chronic skin conditions are available from birth, while others show up all of a suddenly later in life. The reason for these disorders isn't constantly known. Numerous permanent skin disorders have compelling treatments that empower broadened times of reduction. However, they're serious, and side effects can reappear at any time. Contact dermatitis is a standout amongst the most well-known word related illnesses.

 

  • Track 5-1Diffuse cutaneous systemic sclerosis
  • Track 5-2Becker nevus syndrome
  • Track 5-3Squamous cell carcinoma
  • Track 5-4Actinic keratosis

Anaesthesia is getting to be more secure over the previous decades. This procedure was driven by the advancement of new analgesics and the foundation of present day safety ideas. Then again anaesthetists advantage from significant pathophysiological knowledge and the skill of making an interpretation of patient particular conditions into individual perioperative hazard. But in case of rare diseases if there should be an occurrence of it, this idea is frequently inadequate with regards to particular knowledge about the patient conditions and its significance for anaesthesia.

 

  • Track 6-1Congenital diaphragmatic hernia
  • Track 6-2Cerebrotendinous xanthomatosis
  • Track 6-3Larsen syndrome
  • Track 6-4Behcet’s disease
  • Track 6-5Gaucher syndrome

Idiopathic pulmonary fibrosis (IPF) is a prolonged lung disorder considered by thickening, scarring (fibrosis) and stiffening of tissue within the lungs. Affected individuals develop shortness of breath and advanced lung disease. Ultimately, IPF results in life-threatening difficulties such as respiratory failure. Lymphangioleiomyomatosis (LAM) is a rare, advanced lung disease that mostly affects women. Interstitial lung diseases (ILD) are a set of rare lung conditions that effect chronic breathlessness. The problem typically develops over the age of 50 years, and can affect both men and women, and also children.

 

  • Track 7-1Acute interstitial pneumonia
  • Track 7-2Chronic granulomatous disease
  • Track 7-3Juvenile dermatomyositis
  • Track 7-4Kartagener syndrome
  • Track 7-5Mixed connective tissue disease

The most widely recognized rare condition that involves in obstetrical is Gestational trophoblastic disease (GTD), a rare group of diseases that develop in the uterus during an abnormal pregnancy. It arises when abnormal cells or tumours propagate in the tissues that are made following conception that would normally surround the embryo and develop into the placenta. The tumours are denoted to as "moles." Most GTDs are gentle, or noncancerous, however some forms may improve into cancer and spread to other parts of the body. All methods of the disease can be treated and in most cases alleviated.

 

  • Track 8-1Use of vital statistics in obstetrics
  • Track 8-2Hyperemesis gravidarum
  • Track 8-3Polymorphic eruption of pregnancy
  • Track 8-4Psychosocial and environmental pregnancy risks
  • Track 8-5Caesarean hysterectomy

Oral-facial-advanced syndrome (OFDS) is an umbrella term for no less than 10 obviously unique hereditary disorders that are described by irregularities and flaws in the improvement of the structure of the oral cavity including the mouth, tongue, teeth, and jaw; the advancement of the facial structures including the head, eyes, and nose; and the fingers and toes; alongside varying degrees of mental disorder. The introduction of signs and side effects is to a great degree varied, making determination difficult. OFDS type I is the most widely recognized of these disorders, and it is very rare.

 

  • Track 9-1Odontoma dysphagia syndrome
  • Track 9-2Tricho-dento-osseous syndrome
  • Track 9-3Oral submucous fibrosis
  • Track 9-4Bamforth syndrome
  • Track 9-5Dentin dysplasia sclerotic bones

At the point when the typical method of blood cell development and advancement separates, certain cell comprises can be delivered in abnormally large amounts or in abnormal form. This offer arises to blood diseases.  There are many conditions affecting the human haematological system including anaemia-type red blood cell conditions, white platelet dysfunctions related with different malignancies, immuno-disorders, platelet-based variations that influence coagulation, and plasma-based issues (e.g., haemophilia A and B, von Willebrand disease).

 

  • Track 10-1Acute myelomonocytic leukaemia
  • Track 10-2Abdominal aortic aneurysm
  • Track 10-3Acute erythroid leukaemia
  • Track 10-4Chediak-higashi syndrome
  • Track 10-5Blastic plasmacytoid dendritic cell

Aging is a combination of degenerative changes that happen in living beings that do not have the capacity to constantly recover. Age is a main risk factor for most collective neurodegenerative diseases.  The most widely recognized rare condition that involves in Aging is Progeria, an extremely rare autosomal dominant genetic disorder in which symptoms approaching characteristics of aging are revealed at a very early age. Progeria is one of several progeroid syndromes. Those born with progeria typically live to their teenager to early twenties. It is a genetic condition that happens as a new mutation, and is hardly inherited, as carriers generally do not live to reproduce. Studies have demonstrated that 37.5% had conventional life expectancy, 25.7% potentially lethal during childbirth or before 5 years of age, 36.8% reduced life expectancy.

 

  • Track 11-1Premature aging syndromes
  • Track 11-2Mysterious conditions
  • Track 11-3Kallman syndrome
  • Track 11-4Reactive attachment disorder
  • Track 11-5Terminal leukodystrophy

The phrase metabolism related to all the chemical reactions taking place in the body to convert or use energy.  Catalysts assume a key part in a large number of these procedures and changes in their capacity, subsequently, of hereditary transformation can prompt issues in these pathways. Inherited metabolic disorders are genetic conditions that result in metabolism problems Metabolic changes can give incredible assorted qualities of signs and manifestations like episodic illness-anorexia, vomiting, and gastric metabolic disorder, lethargy, cardiomyopathy, muscular, gastrointestinal. Drug treatment is utilized to treat metabolic rare conditions; however this represents an oversees challenge to community and hospital pharmacists as they are not accessible in an appropriate formulation for administration.

 

  • Track 12-1Cerebral folate deficiency
  • Track 12-2Fatal infantile encephalomyopathy
  • Track 12-3Hepatic lipase deficiency
  • Track 12-4Metachromatic leukodystrophy
  • Track 12-5Peroxisomal biogenesis disorders

Numerous rare diseases affect the kidneys and the urinary system, some inadequate to the urinary system itself (e.g., membranous nephropathy), and some part of a condition (e.g., Alport syndrome) that causes additional symptoms in other organ systems. With varying degrees of severity, these diseases can affect different parts of the urinary system (kidney glomerulus, kidney tubules, urinary tract, etc.) causing numerous symptoms.
Several of these diseases occur in the context of inflammation and autoimmune disease (e.g., Goodpasture syndrome). Many rare types of vasculitis can also cause major kidney problems (e.g., Henoch-Schonlein purpura nephritis). The most widely recognized rare condition that involves the genitourinary System is Emphysematous cystitis (EC), a rare infection of the urinary tract that effects in gas production in the bladder. It is more common in diabetic and female patients, and can be associated with more serious complications, including pyelonephritis.

 

  • Track 13-1Amyopathic dermatomyositis
  • Track 13-2Congenital erythropoietic porphyria
  • Track 13-3Cranioectodermal dysplasia
  • Track 13-4Erytoietic protoporphyriahrop
  • Track 13-5Multicentric osteolysis nephropathy

Rare musculoskeletal diseases have diverse symptoms. Some cause muscle weakness or pain, sometimes affecting only specific muscle groups, while others cause skeletal deformation or weakness. In many conditions, other body systems are also affected. Often, symptoms can begin gradually but then worsen as muscles or bones become permanently damaged. The most widely recognized rare conditions that involve the musculoskeletal system are Duchene muscular dystrophy; arise from genetic problems in the muscles themselves, while others involve the nerves, motor neurons, or the bones. A correct diagnosis may come from a neurologist, rheumatologist, orthopaedist, or endocrinologist, numerous physicians and allied health professionals can be of great service in managing a musculoskeletal disease.

 

  • Track 14-1Carpal tunnel syndrome
  • Track 14-2Cystic angiomatosis
  • Track 14-3Focal dermal hypoplasia
  • Track 14-4Endosteal hyperostis
  • Track 14-5Osteoporosis-pseudoglioma syndrome

The majority of rare diseases affect children, most of whom have a primary genetic source for their condition. However, making a molecular diagnosis with current technologies and knowledge is often still a challenge. Paediatric genomics is rapidly developing field but it is not fine developed, that challenges this problem by integrating next-generation sequencing technologies, especially whole-exome sequencing and whole-genome sequencing, into research and medical workflows. This challenging multidisciplinary approach, shared with the rising accessibility of population genetic data variation, has already resulted in an increased discovery rate of connective genes and in developed diagnosis of rare paediatric disease. Substantially, for affected individual family members, a good and systematic understanding of the genetic basis of rare disease translates to more perfect prognosis, management, surveillance and genetic advice; stimulates research into new therapies; and enables provision of better support.

 

  • Track 15-1Juvenile batten disease
  • Track 15-2Improving diagnosis and treatment for severe anaemia
  • Track 15-3Duchenne muscular dystrophy
  • Track 15-4X-linked lymphoproliferative disease
  • Track 15-5Sanfilippo type B

Despite the fact that various endocrine system disorders, for example, diabetes, thyroid, polygenic disorder and osteoporosis are among the most extreme normal and understood therapeutic conditions, a few patients who have been referred to an endocrinologist will require treatment for a rare disease. Number of these rare diseases is risky and rapid myriad Sequelae and secondary diseases. Gland-based hormonal imbalances ensuing in progressive abnormalities and related difficulties are among these diseases, weight regulation issues and physical abnormalities and frequent oncogenic and non- oncogenic endocrine-related tumour disorders.  Rare endocrine diseases can hypothetically impact any organ system, on the other hand.

 

  • Track 16-1Acquired generalized lipodystrophy
  • Track 16-2Central nervous system germinoma
  • Track 16-3Diencephalic syndrome
  • Track 16-4Familial lipoprotein lipase deficiency
  • Track 16-5Granulomatous hypophysitis

The most widely recognized inherent heart defect related to Kounis syndrome might be an episode of angina or even a heart attack activated by an allergic reaction. The syndrome was named later Professor Nicholas G Kounis, a Greek cardiologist, who initiate that the syndrome is caused by chemical substances, for example, histamine, which are discharged by the body during an allergic reaction. These chemicals compounds cause the arteries that supply the heart with blood to go into fit, in this manner constraining the blood stream and causing angina-like chest pain. Torsade de pointe is a risky anomalous heart rhythm that can ready sudden cardiovascular death.

 

  • Track 17-1Tetralogy of fallot
  • Track 17-2Kawasaki disease
  • Track 17-3Takotsubo cardiomyopathy
  • Track 17-4Barlow’s syndrome
  • Track 17-5Kounis syndrome

An Orphan drug might be a medicative item produced for the treatment of an rare disease. Analysis and the treatment of a rare disease wind up complex due to the conditions that various health care providers may have confined contribution with the recognizable proof of the specific disease condition with the diagnosis of rare diseases. Biopharmaceutical specialists in the field have used new advances and the creating investigative appreciation of numerous rare diseases to create and analyse remarkable treatments in the course of the most recent 10 years. In 2015 alone, about half (47%) of novel latest drug approvals were for rare diseases. Numerous blockbuster medications will lose their eliteness in next 5 years. In this way, the current economic situation in addition to the tremendous generic competition moved the focal point of pharmaceutical organizations from the basic medicines to the new business model — niche busters, also called orphan drugs.

 

  • Track 18-1Orphan drug act
  • Track 18-2Clinical development challenges
  • Track 18-3Orphan medicinal products regulation
  • Track 18-4Economic considerations of orphan drugs
  • Track 18-5Challenges in assessing clinical relevance and cost effectiveness

The Orphan Drug gives orphan remaining to medication and biologics that area unit illustrated as those gathered for the safe and effective treatment, assignment or impedance of rare diseases that affect less than two hundred people inside the U.S., or that affect more than two hundred people anyway don't appear to be relied upon to recoup the costs of creating and promoting a treatment drug. The objective of the Orphan store Clinical Trials Grants Program is to support clinical improvement of stock to be utilized in rare diseases or conditions. Last, the paediatric Medical Device Safety and Improvement Act of 2007 (Public Law 110-85) licenses HDE approved devices expected to be utilized in paediatric patients or in an exceedingly paediatric populace and affirmed.

 

  • Track 19-1Congenital aplastic anaemia
  • Track 19-2Pulmonary arterial hypertension
  • Track 19-3Urea cycle disorders
  • Track 19-4Developments in regulating orphan drug approval
  • Track 19-5Cost of drug development

Work in the course of recent years has brought about the identification of qualities in charge of ~50% of the evaluated 7,000 rare monogenic infections and it is anticipated that the majority of the rest of the disease causing qualities will be distinguished constantly 2020 and likely sooner. This checked acceleration is the aftereffect of dramatic improvements in DNA-sequencing innovations and the related examinations. We scrutinize the fast development of rare-disease hereditary analysis and effective techniques for quality identification. We feature the effect of discovering rare-disease-causing genes, from clinical diagnostics to bits of knowledge picked up into biological mechanisms and common diseases. Last, we investigate the expanding therapeutic opportunities and difficulties that the subsequent development of the ' Atlas ' of human hereditary pathology will bring.

 

  • Track 20-1Genome sequencing- revolutionary break through for rare diseases
  • Track 20-2Future detection of rare genetic diseases
  • Track 20-3Potential advancements in therapeutic development
  • Track 20-4Opportunities to accelerate discovery research
  • Track 20-5Development of new drugs for rare diseases
  • Track 21-1Laryngeal papillomatosi