Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym: ...

Sanofi's Genzyme unit has moved a step closer to securing EU approval for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after scoring a positive ...

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Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder; a new report said that plasma lyso-sphingomyelin levels can be used to not only diagnose the rare disease but also predict ...

Positive opinion is based on data from clinical trials which showed improvement in lung function Positive opinion is based on data from clinical trials which showed improvement in lung function The ...

ASMD is an extremely rare, progressive genetic disease with significant morbidity and mortality, especially among infants and children, as many pediatric patients will not survive to adulthood. Signs ...

Sanofi’s Xenpozyme (olipudase alfa-rpcp) is now the only approved treatment for acid sphingomyelinase deficiency. Sanofi announced on Aug. 31, 2022 that FDA approved its drug, Xenpozyme (olipudase ...

The journey to effectively manage and address any medical condition begins with awareness. Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick disease types A, B, and A/B, is one such ...

(RTTNews) - French drugmaker Sanofi (SNYNF, SNY) announced Monday that the Japanese Ministry of Health, Labor, and Welfare or MHLW has granted marketing authorization for Xenpozyme (olipudase alfa) to ...

A recent review examined the significant physical, social, emotional, and financial impacts of acid sphingomyelinase deficiency (ASMD) on patients and caregivers from an overall lifestyle standpoint ...

SAN FRANCISCO & SCOTTSDALE, Ariz.--(BUSINESS WIRE)--PicnicHealth, the venture-backed startup giving patients unprecedented access to their medical records and the ability to contribute to scientific ...