I have a disease called Pellagra. Does anyone else on here have the same disease as me?




I was diagnosed with pellagra over a year ago. It is such a rare disease that it seems like I am the only person I know that has it or has even really heard of it. The doctor that diagnosed me with the disease only knew of it because he was from another country where it was more common. But in the united states it is very unseen. I would like to know if anyone else has it to.

If you have Pellagra, let me know. I hate feeling like the only person with this disease.

Also note that if you do not have this disease or do not know what it is please do not reply. THANK YOU!

Doenças Raras, Rare Diseases




Informativo sobre três doenças raras, Ictiose Harlequin, Neurofibromatose e Síndrome de Proteus. Information on three rare diseases, Harlequin ichthyosis, neurofibromatosis and Proteus syndrome

Dr. Marlene E. Haffner, former Director of the Office of Orphan Products Development at the FDA




Dr. Marlene E. Haffner, former Director of the Office of Orphan Products Development at the FDA and now Executive Director of Regulatory Affairs at Amgen talks to EURORDIS during the 5th European Conference on Rare Diseases in Krakow Poland in May 2010 about the role of Patient Groups in Innovation within the drug development process

Progeria (Part 2)




Very sad story. Watch & learn. No copyright infringement intended.

Sonja Brache, Marshall Smith syndrome, MSS Research Foundation




(In het Nederlands) Sonja was a participant in the European Conference on Rare Diseases (ECRD) Krakow 2010. Sonja is a member of the MSS Research Foundation in the Netherlands.

Angiosarcoma Prognosis – Factors That Affect Survival



Angiosarcoma is a rare malignant neoplasm; which proliferates fast, infiltrates anaplastic cells from blood vessels, and lines irregular spaces filled with blood. The term angiosarcoma is applied to all endothelial vascular tumors that are progressive, destructive, and aggressive. The disease rarely occurs in large vessels and is very common in the skin and body organs. The clinical behavior is difficult to predict and the symptoms are not specific enough or late to appear.

The prognosis is generally poor and depends much on these two factors:

Investment in Health & Chemicals Sector in UK



The UK has one of the fastest growing and strongest Biotech and Pharmaceuticals sectors in the world. The growth of this sector is driven by its rich pool of skills and talent. A strong academic base, government support for R&D through investment and tax credits attract leading companies to the UK. The UK life sciences sector enjoys an outstanding rate of success. Twenty-five of the world’s 100 top selling medicines were discovered and developed in the UK.

Infrastructure for the Biotech and Pharmaceuticals sector

The UK offers superior educational institutions and training and research facilities that help develop and support clinical research. The UK is home to many postgraduate degree granting institutes that specialize in regulatory and clinical affairs. The UK has made a major investment of more than 200 million pounds recently in R&D in the UK biotech and pharmaceuticals sector which has boosted the capacity and capability of early phase clinical studies and experimental medicine research.

The UK Clinical Research Network, formed in 2005, provides a performance managed infrastructure to industry sponsored clinical trials, investigator initiated trials, and well designed studies. This infrastructure support is given in association with National Health Services. UKCRN research includes a series of topic specific and comprehensive research across the network spread in the UK. The research covers a full range of medical conditions, including rare diseases. The UKCRN also works with biotech, pharmaceuticals and devices to facilitate and streamline commercial research, and industry sponsored trials.

Stable Regulatory environment

The UK provides a balanced regulatory environment to the sector. The stable environment takes into consideration human and environmental safety and at the same time encourages innovation and entrepreneurial efforts. The government is taking steps to streamline the regulatory environment which includes streamlined systems for research permissions and approvals. These amendments will speed trial start times. UK health governments have the right strategies in place to make the UK the best place in world for health research, innovation and development. The government has also increased its funding to the sector which will reach 1.7 billion pounds per year by 2010-2011. The world class scientific institutions, strong research base and outstanding record in product development are attractive for investors to invest in UK’s Biotech and Pharma sector.

Chemicals and Process Industries

UK chemicals and process industries have firm foundations due to the presence of an excellent infrastructure, extensive technical expertise, support network and skills. It is one of the largest manufacturing sectors, accounting for 11% of total revenue from manufacturing sector.

The growth of this sector is based on core strengths of science, innovation and research and development. The UK supports four key process and chemical industries:

• Base and Petrochemicals
• Fine and Specialty Chemicals
• Biotechnology
• Pharmaceuticals manufacturing

UK chemicals and process industries gain advantages from a range of facilities to produce sustained growth in the sector in the future. The chemical industry has laid its strong foundations based on productive relationship between public and private sector companies, local academic institutions and technology driven industry experts. The 10 million pound, 21st century training center, National Skills Academy for the Process Industries provides training and world class opportunities to new entrants and existing workers to update and learn new skills. This training facility will play an important role in creating a new workforce for the future. The government has doubled its investment in R&D which has shown impressive results. The government also offers tax incentives in R&D to companies based in UK.

The UK intends to attract new investment in pharmaceutical and specialties investment depending upon regional excellence. UK is one of the world’s leading chemical producing nations that continue to grow with exports, outstanding level of growth, productivity performance and foreign direct investment.

Rare Diseases Day – Tuberous Sclerosis Complex (TSC)




Video clip from ABC7 Chicago of Ed Sitar being interviewed regarding Rare Diseases Day and Tuberous Sclerosis Complex (TSC)

National Organization for Rare Disorders 25th Anniversary




President Ronald Reagan signed the Orphan Drug Act in 1983, bringing new hope to the 25 million Americans with rare diseases. Throughout 2008, NORD be celebrating the 25th anniversary of this very successful legislation and of the founding of NORD.

What is your thought on Christian faith healers?




and this… you can call me a hard agnostic because I haven’t been able to rule out the though of an afterlife so much a god…

I attended one church a couple of years (about ten years ago) … they frequently had famous faith healers which I never new of… and one came and said some things that he obviously could see single mom kids.. some figurative statement but then he waved his hand in my face and I PASSED OUT… for a while…

Went home told my sisters.. and they were 11 and 13 and didn’t believe me … the youngest had a rare disease and I conviced her to go with me next week (dermatomyositis and was currenly dying from necrotic cap. arteries, veins.. etc…..) …..she couln’t take the chemo because her liver was bad for no known reason… short story waved his hand over her liver and she passed out and the next week she started chemo because her liver was good and soon went into remission..
Do you think maybe it’s drugs on there hands… or even a mental power??
Apparently it didn’t last because she is about to be 20 … and has been out of remission lots of times… but her liver can take the drugs…..

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