Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…
Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…
Do you know the symptoms of ovarian cancer? If someone asked you to list the symptoms right now, could you? If you were experiencing symptoms of ovarian cancer yourself, would…
The first new treatment approved for amyotrophic later sclerosis (ALS) has generated a lot of excitement and enthusiasm in the ALS community since its approval in May of 2017, but…
Information in a Specialty Pharmacy Times report indicates that immunotherapy combined with chemotherapy may be an effective option for patients with drug-resistant multiple myeloma who have relapsed. Multiple myeloma is…
According to a story from dailyhealthneeds.com, long term, regular use of protein pump inihibitors (PPI) can majorly increase the risk of contracting stomach cancer. PPIs are commonly used to treat…
Many cancer and blood specialists will not have encountered systemic mastocytosis. A new drug targets the underlying defect of the disease and will help put doctors on the lookout. Keep…
Out-of-pocket expenses for rare disease are astronomical, and the Ministry of Health (MOH), is taking time to find a more positive way to help benefit children and their families burdened…
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