Over 10,000 rare diseases have been identified globally, a large majority of which are genetic in origin. But despite the scope of rare diseases, treatment can still be difficult to…
David Guy, parent of Kaila who was diagnosed with acid sphingomyelinase deficiency (ASMD), recounts his initial shock when he was told of Kaila's rare disease. David and his wife…
According to a story from Globe Newswire, olipudase alfa (marketed as Xenpozyme) has been approved by the European Commission for the treatment of non-CNS related symptoms of acid sphingomyelinase deficiency…
According to a story from Globe Newswire, The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently announced a positive opinion in regards to the experimental…
In the past, there has never been an approved therapeutic option for those with acid sphingomyelinase deficiency (ASMD). This meant that there was a huge unmet need within this patient…
Written By Joslyn Crowe Caring for someone with a rare disease, especially a child or a family member, can be a difficult and emotional experience. Many people become a caregiver…
In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…
Purnell was Taylor and Sam Sabky’s first child, and they couldn’t have been more excited for his arrival and the pregnancy and delivery happened without any major issues. For the…
Dr. Justin Hopkin's son Garrett was born happy and healthy, but when he was around four to six months old, his parents began to notice some problems. Garrett was having…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
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