Rare Disease in the News
Boys plans to outpace rare disease in Flying Pig Marathon
He is battling a rare and fatal disease known as Duchenne muscular dystrophy, although you wouldn't know it by his friendly and smiling demeanor. Come early Sunday morning, he will have 80 helping hands and many more hearts powering a wheelchair as ...
Shire profit and revenue up on rare disease drugs
Shire PLC said profit and revenue climbed in the first quarter, thanks to strong sales of several rare disease medicines and its blockbuster pill for attention-deficit hyperactivity disorder, or ADHD. The Dublin-based drugmaker said net income rose 2 ...
Shire chief shrugs off shareholder protests
Shire - Q1 sales up 17%
Shire Quarterly Profit Tops Estimates as Drug Sales Soar
Rare disease gene has a key role in chronic hepatitis C infection
ABHD5 stood out because mutations in the gene cause a rare human genetic disease called Chanarin-Dorfman syndrome (CDS) that causes defects in lipid storage, and also because the earlier tests suggested that the gene had an interesting role in lipid ...
Young girl with rare disease dances alongside Brooklynettes on 'Rachael Ray Show'
Audrey Nethery is a 7-year-old girl that hails from Louisville, Kentucky. At 2 months old she was diagnosed with a rare, inherited bone marrow failure syndrome called Diamond Blackfan Anemia. But that doesn't stop her from dancing. On Wednesday, she ...
WATCH: Girl with rare disease gets down with Nets dancers
WATCH: Kentucky Girl with Rare Disease Makes Adorable Appearance on Rachael Ray
Currently, there's no cure for the disease – an inherited bone marrow failure syndrome – but the family has set up a GoFundMe account and hope to raise $50,000 to help find a cure. All proceeds from the crowd funding site go directly to the DBA foundation.
Vatican cell conference opens with focus on kids, rare disease
In her opening remarks, the President of Stem For Life, Dr. Robin Smith, pointed to the growing range of therapies currently under study for the treatment of cancer, autoimmune disorders and rare diseases. The first in the series of conferences was ...
CORRECTING and REPLACING The Vatican Hosts Third International Regenerative Medicine Conference
Sanford Health Selected for Global Cell-Therapy Event at Vatican
Mother creates foundation in daughter's name who died of rare disease
KVIA El Paso
The Aileen Burkett HLH Foundation is still in the works. Herrera said it's a way to cope with her loss and make parents aware. "In this world, you are here to serve," Herrera said. "I want people to know about this disease so they don't go through this ...
Cherry Hill Courier Post
TLC to feature SJ man with rare disease
Cherry Hill Courier Post
GLOUCESTER TWP. - At first, Kenny Krips thought the emails asking him to appear on a new television documentary series were a scam. But after several phone calls with the show's producers, the 45-year-old Glendora resident agreed to appear on TLC's ...
Pope advocates for 'globalization of empathy' in curing rare diseases
“It is fundamentally important that we promote greater empathy in society, and not remain indifferent to our neighbor's cry for help, including when he or she is suffering from a rare disease,” the Pope said April 29. While it's not always possible to ...
Pope Francis on rare diseases: patients should not feel abandoned
Biden gets papal blessing for his global war on cancer
Pope's Address to Regenerative Medicine Conference
TLC unites NJ man with woman who shares rare disease
When you are the one in 600,000 that are born with a rare disease, life can feel a bit lonesome at times. Glendora resident Kenny Krips was born with Lamellar Ichthyosis — a rare condition that causes the skin to grow ten times faster than normal ...
Jersey Man With Lamellar Ichthyosis Featured In Documentary: What Is This Rare Skin Disease?
'Two In A Million': Sneak Peek of Bobbi and Kenny's Story of Living With a Rare Disease (VIDEO)
South Jersey man to be featured on TLC show 'Two in A Million'