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The only patient in an FDA-sanctioned clinical trial assessing a CRISPR-based therapy for a rare form of Duchenne muscular dystrophy has died, according to study sponsor Cure Rare Disease.
With diagnostics for Alzheimer’s already approved and therapeutics in late-stage clinical trials, the next hurdle is to translate these advances into clinical practice. The Davos Alzheimer’s Collaborative is working on it.
The Institute for Clinical and Economic Review (ICER) concluded that even at a hefty lifetime cost of $2.5 million, gene therapies for hemophilia A and B are worth it.
CRD’s CRD-TMH-001 is expected to upregulate an alternate form of the dystrophin protein with CRISPR technology and stabilize or reverse the symptoms associated with DMD.
GSK launched the “Target the Future” initiative to improve the care of patients diagnosed with multiple myeloma, the third most common form of hematological cancer.
The buyout comes on the heels of promising Phase I/II results from GTX-102, an antisense oligonucleotide candidate being developed to treat Angelman syndrome.
In a study conducted by researchers at Stanford University, three pediatric patients with a rare genetic disease that often results in kidney failure received kidney transplants.
The Tisch MS Research Center of New York has been researching the concept of using cell therapy as a therapeutic strategy to promote repair and regeneration in certain patients with MS.
Portland-based Children’s Cancer Therapy Development Institute is discovering more targeted drugs for childhood cancers and driving them into clinical development with industry partner Artisan Biopharma.
BioSpace looks at the challenges faced by the Latinx community in the life sciences, from the origins of the challenges, to educational gain and the importance of mentorship.
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