New Stem Cell Treatment for ALS May Slow Disease Progression.Smoking marijuana could be bad for your heart, but there is an unusual remedy.More Photos Categories Categories Archives Archives Top Read Posts Making a Deposit in the Bank: Using Stem Cells from Children with Rare Diseases to Find New Treatments.How Research on a Rare Disease Turned into a Faster Way to Make Stem Cells.Rare Disease Day (2016), A Chance to Raise Awareness and Hope.And those suffering from rare disease are very clearly people in need. We are in the business of accelerating treatments to patients with unmet medical needs. We are not in the business of making value judgements about who has the greatest need. But the lessons we learn in developing treatments for a rare disease can often lead us to treatments for diseases that affect many millions of people.īut numbers aside, there is no hierarchy of need, no scale to say the suffering of people with Huntington’s disease is any greater or less than that of people with Alzheimer’s. Some people might ask why spend limited resources on something that affects so few people. For example one drug, Defactinib, has shown some promise in inhibiting the growth and spread of cancer stem cells – these are stem cells that can evade chemotherapy and cause patients to relapse.” Right now, treatment for mesothelioma includes the usual combination of chemotherapy, radiation, and surgery, but researchers are looking at other approaches to see if they can be more effective or can help in conjunction with the standard methods. Symptoms can have a very sudden onset, even though the person may have been exposed decades prior. The late stage discovery of mesothelioma is often what causes it to have such a high mortality rate. However, it was found that once asbestos crumbled and became powder-like, the tiny fibers could become airborne and be inhaled and lodge themselves in lung tissue causing mesothelioma. Having a home with asbestos insulation, ceiling tiles, and roof shingles meant that the house was safer. Malignant mesothelioma is among the 95 percent that doesn’t have a treatment or cure.Īsbestos has been used throughout history in building materials because of its fire retardant properties. Rare diseases affect 300 million people worldwide, but only 5% of them have an FDA approved treatment or cure. Groups like the Mesothelioma Cancer Alliance and the Mesothelioma Group are adding their voices to the cause to raise awareness about mesothelioma cancer, a rare form of cancer caused by exposure and inhalation of airborne asbestos fibers To see how you can help support the Rare Disease Day efforts this year, click here. They use their specific perspectives to raise their voice, share their story, and shed light on the areas where additional funding and research are most necessary. It has long been known that the best advocates for rare diseases are the patients themselves. Awareness for rare diseases is extremely important, especially among researchers, universities, students, companies, policy makers, and clinicians. Getting involved for Rare Disease Day means taking this message and spreading it far and wide. For the year 2017 it was decided that the focus would fall on “research,” with the slogan, “With research, possibilities are limitless.” The campaign works to target the general public as well as policy makers in hopes of bringing attention to diseases that receive little attention and funding. This is a day dedicated to raising awareness for rare diseases that affect people all over the world. “February 28th marks the annual worldwide event for Rare Disease Day. Today we have asked Emily Walsh, the Community Outreach Director at the Mesothelioma Cancer Alliance, to write about the efforts underway to raise awareness about rare diseases, and to raise funds for research to develop new treatments for them. There is still so much work to do.Īnd all around the world people are doing that work. It’s a good start but clearly, with almost 7,000 rare diseases, this is just the tip of the iceberg.
Since then we have funded 13 new clinical trials including four targeting rare diseases ( retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s disease, and Duchenne’s Muscular Dystrophy). One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases.
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