Bethesda, Md. — HYFTOR® (sirolimus topical gel) 0.2%, indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and pediatric patients 6 years of age and older, is now covered by Medicare in Texas, Florida, New York, and California, among other states. Nobelpharma, a pharmaceutical...
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London, UK – Patients with rare diseases could benefit from a “revolution” in clinical trials that could see one-stop studies designed that provide robust results even with small numbers of participants. The CAPTIVATE node is part of the recently introduced UK Rare Disease Research Platform established as part of a...
Last month, Jessie Jackson’s friends and family sang her happy birthday a little early. Jessie doesn’t turn 30 until Dec. 12, but for the past five years on the anniversary of her diagnosis, her parents have hosted a fundraiser. They weren’t taking any chances. Jessie inherited a rare genetic condition called GM1...
Rare diseases are a serious public health concern in India, with an estimated burden of about 80 to 96 million cases reported annually. Moreover, 70-80% of rare diseases are of genetic nature and thus are asymptomatic. Here are their causes, signs, symptoms and treatment
February 25th was a busy day at the oval and the Recreation Complex. Many people showed up to give their support for the Canadian Organization for Rare Diseases (CORD). One of our own little citizens, 15-month-old Iris Mae McKechnie was diagnosed with infantile onset pompe disease and as a result...
Rare Diseases South Africa (RDSA) held its annual Denim Walk at Walter Sisulu Botanical Garden on March 4. Family, friends, survivors, and sufferers turned lush green lawns into a sea of white cotton and blue denim. South Africa has just short of four million suffering from rare genetic and congenital...
There are around 7,000 rare diseases that we know of today. Each of these conditions only affects a small number of people, but collectively, they impact a large population – estimated at 30 million people in the EU. Most rare diseases are severe and life-threatening. However, 95 percent of people...
DURHAM, N.C. — Children born without thymus glands have given Duke University Medical Center researchers a rare opportunity to watch as a new immune system develops its population of infection-fighting T-cells. Researchers led by Thomas Kepler, Ph.D., Division Chief of Computational Biology, tracked three young patients after thymus tissue transplantation...
Georgia Uphill is four years old but has the mental age of an 18-month-old. She does not speak and needs help to walk. She hyperventilates and easily becomes anxious. Georgia, who lives in St Thomas, Exeter, with her mum Katie, dad Ben and seven-year-old brother William, has Rett Syndrome. She...
London, UK – Research led by Claudia Cooper, Professor of Psychological Medicine at Queen Mary University of London, shows that a new therapy, NIDUS-Family, helps people with dementia and their family carers attain their personal goals. The NIDUS-family package of care and support focuses on practical changes people can make, with sessions designed around the specific priorities of the...