Hosted on MSN1y
Larimar lays out FDA approval plan for Friedreich’s ataxia drug
Nomlabofusp is a protein replacement therapy designed to deliver frataxin protein to the cells of people with Friedreich’s ataxia. The drug was generally well tolerated. One participant in the ...
Hosted on MSN1mon
SLDB Stock Up as FDA Clears IND for SGT-212 in Friedreich's Ataxia
LRMR believes that nomlabofusp has the potential to be the first frataxin protein replacement therapy for patients with FA. Solid Biosciences currently carries a Zacks Rank #3 (Hold). A better ...
Yahoo Finance19d
Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA. Fast Track designation is granted to ...
Pharmabiz1mon
US FDA clears Solid Biosciences’ IND application for SGT-212 to treat neurologic & cardiac manifestations of Friedreich’s ataxia
Friedreich’s ataxia is a highly complex, multisystem disease that presents distinct challenges for drug development, in part because frataxin is a protein that requires: precise expression levels to ...
Nasdaq1mon
SLDB Stock Up as FDA Clears IND for SGT-212 in Friedreich's Ataxia
LRMR believes that nomlabofusp has the potential to be the first frataxin protein replacement therapy for patients with FA. In the past 60 days, estimates for Puma Biotechnology’s 2025 earnings ...
Pharmabiz22d
Solid Biosciences’ AAV-based gene therapy candidate, SGT-212 for Friedreich’s ataxia receives US FDA fast track designation
designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA. Fast Track designation is granted to products ...
Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation - CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: ...
Business Insider24d
Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
- Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation - CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE ...