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

Dr. Scott Loiler The National MPS Society is pleased to announce the appointment of Dr. Scott Loiler as its new Chief Scientific Officer (C ...

Learn more about whether Ultragenyx Pharmaceutical Inc. or Vericel Corporation is a better investment based on AAII's A+ ...

Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...

Details of the oral presentations are as follows (all times in PST; * denotes corresponding poster): Title: Hematopoietic stem cell gene therapy for mucopolysaccharidosis type I-Hurler (OTL-203): ...

Denali’s candidate is its DNL12 enzyme replacement therapy (ERT) for mucopolysaccharidosis ... Neurogene has contributed its gene therapy NGN-401 for Rett syndrome, a rare genetic neurological ...

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

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

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

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.