When Kara Roberts learned that she was pregnant with a daughter, she was thrilled. While her pregnancy itself was relatively normal, Roberts learned that her daughter, Scarlett, would need one…
According to a recent article, the parents of a boy with epilepsy were told they would need to prepare for him to have end-of-life care after they were unable…
Earlier this year, the MHRA in the United Kingdom expanded the license for Kaftrio, a treatment option for patients with cystic fibrosis (CF). When this expansion occurred, allowing for the…
In late October 2021, the National Institute for Care and Excellence (NICE) in England recommended Givlaari (givosiran) to treat patients with acute porphyria -- or, more specifically, to treat patients…
According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…
As originally reported in Medscape; many rare disease patients know the frustration of not having any drugs or treatments available for their disease. 95% of rare disease patients have no…
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Dr. David Porteous, a General Practitioner in Bristol, England recently reported to the Bristol Cable, a community-run newspaper, that he does not agree with the extensive list of treatments being…
England’s National Institute for Health and Care Excellence (NICE) recently approved three new thyroid cancer treatments for their National Health Service (NHS). The new treatments provide a wider variety of…
England’s National Health Service has just approved a new drug for the treatment of breast cancer. While the drug is most effective in specific cases, advocacy group Breast Cancer Now…
Tessa Jowell, former Labour party cabinet minister, delivered a moving speech to the UK House of Lords last week. The speech largely concerned her battle with glioblastoma, a rare brain cancer.…
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