Ultragenyx reports positive data on gene therapy for rare disease
NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE), a $4.08 billion market cap biotechnology company with 27% revenue growth in the past year, today unveiled new data indicating ...
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What happens when a rare disease drug seems to work, but the health care system doesn’t?
Cristina Brennan can hear the clock ticking. At the end of the year, her 12-year-old son Tristan will no longer have free ...
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UAE: Footballer battles rare brain cancer, returns to field after 5 years
Despite the gruelling treatments, Khaled refused to lose hope and drew strength from his faith and the unwavering support of friends, fellow survivors ...
New Bedford pastor nearly paralyzed from a rare disease. He's made a miraculous comeback.
Christian Fellowship Center Lead Pastor Brian Dube is making a miraculous recovery from a case of near-fatal paralysis from a ...
Science Daily11h
Breakthrough in childhood brain cancer research could heal treatment-resistant tumors, keep them in remission
Research has shown that a potential new targeted therapy for childhood brain cancer is effective in infiltrating and killing tumor cells in preclinical models tested in mice. The novel drug CT-179 was ...
Science Daily13h
Research discovery halts childhood brain tumor before it forms
A research team has identified a critical event driving tumor growth in a type of medulloblastoma -- and a way to block it.
I’ve had skin cancer twice and two brain tumours and I’m 18 – I live in fear of dying sooner than a normal person
A TEEN who’s been diagnosed with melanoma twice and had two brain tumours removed, all before the age of 18, has a rare ...
Teenager diagnosed with brain tumours and skin cancer twice - before her 18th birthday
Ruby-Jo Sneed, 18, was diagnosed with a rare genetic chromosome deletion after undergoing genetic testing aged 12 due to her ...
For individuals with this disorder, sleeping for extended periods of time is a haunting reality.
This was highlighted by Dr Sermed Mezher in a recent reel posted on Instagram, “People with KLS experience recurring episodes where they sleep excessively, sometimes up to 20 hours a day, for weeks at ...
Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
Scientists are unraveling the cause behind sudden unexpected death in epilepsy
In the United States, nearly three million adults 18 years and older reported having active epilepsy during 2021 and 2022.