The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

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Announcing a new article publication for BIO Integration journal. Neurologic disorders currently affect approximately 100 ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...

According to research by nova one advisor, the global cell and gene therapy market size was valued at $18.13 billion in 2023 ...

A new case report was published in Volume 16 of Oncotarget on February 5, 2025, titled “Acquired RUFY1-RET rearrangement as a ...

Unresectable canine hepatocellular carcinoma (HCC) has limited nonsurgical treatment options. Sorafenib is a targeted therapy for unresectable canine HCC. However, there are limited reports on the ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.