Annual Congress on Rare Diseases and Orphan Drugs

Rare Disease is a branch of medicine which is an important public health issue and challenge for the medical community. They are called “Health Orphan”. The term “Orphan Drug” refers to a drug or biologics such as vaccine or blood product that treats a rare disease or conditions. Rare Diseases-2016 will be the best platform for all the specialists and super specialists, renowned Scientists, research scholars, students who are working in this field across the globe under a single roof to exchange their knowledge related to Rare Diseases and Orphan Drugs. This international event is an effort to find an alternative for invasive imaging technique against diseases like rare infectious diseases, rare cancer, Morgellons, Renal cell carcinoma, malignant glioma, Multiple myeloma, Hepatocellular carcinoma etc.

“Annual Congress on Rare Diseases and Orphan Drugs” is going to be held on 26-27 Oct, 2016 at Chicago, Illinois, USA. The organizing committee is gearing up for an exciting and informative conference program including plenary lectures, symposia, workshops on a variety of topics, poster presentations and various programs for participants from all over the world. We look forward to having you be part of the event!

Rare Diseases-2016 is going to held in Chicago, a city in the U.S. state of Illinois, is the third most populous city in the United States and the most populous city in the American Midwest, with approximately 2.7 million residents. Its metropolitan area (also called “Chicago land”), which extends into Indiana and Wisconsin, is the third-largest in the United States, after those of New York City and Los Angeles, with an estimated 9.8 million people. Chicago is the county seat of Cook County, though a small portion of the city limits also extends into Du Page County.

There are approximately 7,000 rare diseases, which from a regulatory perspective are defined as those diseases where there are less than 200,000 patients in the US or that affect no more than five in 10,000 of the general population in the EU. “Orphan drugs” are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are “orphans” because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.

For more details- http://rarediseases.conferenceseries.com/

For registration details- http://rarediseases.conferenceseries.com/registration.php

Dr. Ebraheim’s educational animated video describes multiple myeloma.
Multiple myeloma is cancer of the plasma cells. It is malignant proliferation of the plasma cells. Plasma cells are a type of blood cells that produce antibodies. Antibodies are an important part of the immune system that fights infection.
Multiple myeloma cells produce a single protein – Monoclonal protein, M protein. Multiple myeloma occurs more often in males and twice as often in African Americans. It usually occurs in patients older than 40 years of age. Multiple myeloma is the most common primary malignant bone tumor.
The plasma cells secrete osteoclast activating factors such as interleukin 6, macrophages and inflammatory factors in addition to RANKL. Osteoclasts are recruited, activated and they multiple. Osteoclasts absorb the bone. The osteoblast bone formation is suppressed by the tumor necrosis factor (why there is a cold bone scan in multiple myeloma). In about 30%, the bone scan is negative. Get a skeletal survey.
Clinical features: Useful mnemonic to help remember the most common features of multiple myeloma is CRAP, which stands for hypercalcemia, Renal failure, Anemia, and back Pain. Clinical features will be insidious presentation. Bone pain, especially back pain with possible fractures, weakness and fatigue, cord compression, infections and fever. Multiple myeloma can occur in any bone although it occurs more in the hematopoietic bone marrow!
Labs: Sedimentation rate may be elevated. May find renal insufficiency, hypercalcemia, and anemia. Electrophoresis will find a spike in the serum, in the urine, or in both. Serum Protein ElectroPhoresis (SPEP) detects monoclonal elevation of the immunoglobulin and identifies the protein types that are present. Urine Protein ElectroPhoresis (UPEP) – bence jones proteinuria (urine) and light chains (about 95% sensitivity).
What are the tumor markers that indicate a bad prognosis? Increased serum beta1-microglobulin level and a decreased serum albumin.
How do you establish the diagnosis? One major and one minor criteria is needed or three minor criterion.
What are the major criteria? The major criteria are plasmacytoma tissue diagnosis on biopsy, more than 30% plasma cells in the bone marrow, SPEP with great value for IgG or IgA and/or UPEP (Bence Jones Proteinuria).
The minor criteria are 10% – 30% plasma cells in the bone marrow, monoclonal protein in the serum or the urine (SPEP/UPEP – these values are usually lower than in the major criteria), multiple “punched-out” lytic bone lesions, end-organ damage (renal insufficiency, hypercalcemia, and anemia), and lower than normal IgG levels.
Images: Images will usually show “punched-out” lytic lesions throughout the skeleton. Radiolucent lesions or destructive lesions with no sclerosis (skull lesions and vertebral fractures). Osteopenia is common. There will be a negative bone scan. Minimal osteoblastic activity with multiple myeloma due to the tumoral necrosis factor. Skeletal survey can be used. Sometimes a magnetic resonance imaging (MRI) or positron emission tomography (PET) scan may be used. Bone scans are generally not done in cases of multiple myeloma (bone scans will be cold).
Pathology: Sheets of plasma cells, Eccentric nucleus (clock faced), Signet-ring appearance, Perinuclear clearing, Abundant eosinophilic cytoplasm.
Treatment: Usually high-dose chemotherapy. Radiation treatment for progressive lesions, structurally important lesions, or radiation is used to decrease pain. Bone marrow transplant (usually in younger patients). Bisphosphonate decreases bone pain, calcium and the number of lesions. Surgical stabilization is used for prophylactic fixation or prevention of pathological fractures. It can also be used for the treatment of fractures.

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