“In so many ways, [my children] are pioneers, and I could not be prouder of them,” Jillian Arnold tells PEOPLE ...

Xenpozyme is a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy designed to replace deficient or defective ASM. The Food and Drug Administration (FDA) has approved Xenpozyme ™ ...

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: ...

Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomyelinase deficiency “These significant results for olipudase ...

A small cohort of patients with acid sphingomyelinase deficiency (ASMD) showed higher rates of cancer compared with the general population, according to recent findings. Acid sphingomyelinase ...

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 ...

Acid sphingomyelinase deficiency (ASMD) is a progressive lysosomal storage disease caused by autosomal recessive pathogenic variants in the SMPD1 gene encoding acid sphingomyelinase (ASM). In a recent ...

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 ...

ASMD is a rare, debilitating lysosomal storage disease characterized by a deficiency of the enzyme acid sphingomyelinase, which results in the accumulation of sphingomyelin in various tissues of the ...

Please provide your email address to receive an email when new articles are posted on . The Japan Ministry of Health, Labor and Welfare has authorized Xenpozyme for the treatment of adult and ...