Cell cycle re-entry of senescent breast cancer cells after CDK4/6 inhibition (CDK4/6i) constitutes a major source of disease ...

AceLink is advancing a small-molecule treatment option for Fabry disease, which could provide a more convenient alternative ...

Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.

Discover Regional Growth Trends and Shares for Galsulfase, Velaglucerase Alfa, Laronidase, Asfotasealfa, and Other ERT ...

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Ultragenyx announces new safety and efficacy data from its pivotal study of its investigational candidate, UX111, for ...

Carisma Therapeutics Inc. ("Carisma" or the "Company"), a leader in macrophage-focused therapeutics, today announced that Steven Kelly, President and Chief Executive Officer, will participate in the ...

The global enzyme replacement therapy market size was valued at USD 10.12 billion in 2024 and is projected to grow from USD 11.35 billion in 2025 to reach USD 21.29 billion by 2033, exhibiting a CAGR ...

The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...