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Most disease-causing mutations that swap amino acids do so by making proteins less stable
Research shows 60% of missense mutations destabilize proteins, linking protein folding to genetic diseases, including ...
Denali: Despite ALS Primary Endpoint Failure, Possible Path Forward Remains
Denali Therapeutics Inc.'s stock remains stable despite trial setback in ALS. BLA filing for MPS II treatment on track. Click ...
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II)
Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment ...
Hosted on MSN2d
MPs vote on national inquiry into grooming gangs
Labour MPs rejected a Tory demand for a new national inquiry into grooming gangs. The House of Commons opposed a call, ...
GEN4d
Running Up That Hill: An Interview with Carole Ho, Denali Therapeutics
On a recent visit to Washington DC, Ho sat down with GEN Editorial Director Kevin Davies, PhD, to discuss Denali’s progress ...
ITN on MSN2d
MPs vote to reject national grooming gangs inquiry
MPs vote to reject national grooming gangs inquiry in Commons ...
Former Man Utd star tells of dementia fear
A man who has won some of football's highest honours has spoke of his fears he could develop dementia due to heading the ball ...
New agency to boost tackling of infectious diseases to be set up by first half of 2025
The Communicable Diseases Agency has five areas of responsibility, including prevention and response. Read more at ...
Business Insider23d
Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
About UX111 UX111 is a novel in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment that ...
Yahoo Finance23d
Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
enabled us to file our BLA and may unlock the future accelerated approvals of a host of new therapies for these devastating MPS diseases that affect the brain.” Earlier this year, Ultragenyx ...
pharmaphorum4d
Regenxbio expands MPS I gene therapy trial after first look at data
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 ...
Joplin Globe5d
JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy
JCR Pharmaceuticals Co., Ltd. (TOKYO:4552; “JCR”) and Modalis Therapeutics Corporation (TOKYO:4883; “Modalis”) today announced that they have validated the initial proof of concept in a joint research ...