Rare genetic blood diseases
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The road to diagnosis and treatment for patients with rare and genetic diseases is one with very little navigation. With over 7, 000 rare diseases and one in every ten people diagnosed - chances are you know someone, or may even BE someone with a rare disease.
When fewer than 200, 000 patients struggle with a disease- who serves to educate, empower, and lead the sick to the right doctors, the right medications and the fastest road to relief?
Behind the Mystery of: Rare and Genetic Diseases is a series produced with the intention of introducing and uniting patient, physician, and scientist with research, education and opportunity to revolutionize the way our health care system works for the Rare and Genetic Minority.
By partnering with The Global Genes Project, The Balancing Act is able to provide available support to patients diagnosed with rare and genetic diseases, and to give hope with news of clinical trials and new treatments. Together, we can begin a new wave of advocacy and support for the rare community.
The Balancing Act supports and celebrates patient advocacy groups and industry leaders, who inspire connect and guide patients as they move forward through their journey towards treatments and a cure.
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No. But days after this happened we learnedby --
Of a relative who had the same things starting at age 40. The person is still alive at age 88. So that's good. The docs are doing a blood test re: rare genetic diseases. This could shed light on the situation.
But...he is overweight. However, he exercises 4-5 times a week. Hardcore exercise -- things like spin class and 50 mile bike rides and regular 1.5 gym outings. He loves exercise.
Textbook of Orthopaedics, Trauma and Rheumatology
AGTC and SAFC Create Strategic Partnership to Provide Gene Therapy .. — GlobeNewswire
AGTC's lead product candidates, which are each in the preclinical stage, focus on X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are rare diseases of the eye, caused by mutations in single genes, that significantly ..