Sarepta says early filing for DMD gene therapy is back on
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy […]
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy […]
New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have […]
Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test […]
A Duchenne muscular dystrophy drug candidate from Italfarmaco Group has encouraging preliminary data from a pivotal study. The Milan, Italy-based pharmaceutical company says it now […]
Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after […]
Code Biotherapeutics uses synthetic DNA as the foundation for its genetic medicines, which the startup claims offer several key advantages compared to genetic medicines delivered […]
PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing […]
Pfizer now has the regulatory O.K. to resume pivotal tests of its gene therapy for Duchenne muscular dystrophy—but with new limitations and safety measures. The […]
Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the […]
Just days after dosing its first patient in a Duchenne muscular dystrophy trial, US biotech PepGen has pushed the button on an initial public offering […]
The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it […]
Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a […]
The FDA has halted tests of a Pfizer gene therapy for Duchenne muscular dystrophy. The clinical hold is one of several placed on gene therapy […]
A year ago, Swiss biotech Santhera was slashing staff to cut costs after it abandoned lead Duchenne muscular dystrophy (DMD) therapy idebenone. Now, it’s ready […]
Capricor Therapeutics’ bid to find a partner for its Duchenne muscular dystrophy (DMD) cell therapy CAP-1002 could get a boost after final phase 2 data […]
Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy posted encouraging results from a Phase 2 study enrolling 20 patients. But the FDA said a Phase […]
Duchenne muscular dystrophy (DMD) is a rare genetic disorder usually diagnosed in young boys, gradually weakening muscles across the body until the heart or lungs […]
Date: June 28, 2021 Issue #: 1627 Summary: Casimersen (Amondys 45 – Sarepta), an IV antisense oligonucleotide, has received accelerated approval from the FDA for […]
Shots: The P-IIb VISION-DMD study involves assessing the efficacy & safety of vamorolone (2&6 mg/kg/day) vs PBO and prednisone (0.75 mg/kg/day) in 121 ambulant boys […]
Shots: Sarepta reported positive 12wks. expression & safety results from the first 11 participants enrolled in Study SRP-9001-103. The study uses commercially representative SRP-9001 Patients […]
The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Pfizer’s CIFFREO study began in the […]
A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of […]
The FDA decision gives Sarepta Therapeutics its third approved drug for Duchenne muscular dystrophy. The accelerated approval requires the biotech to conduct additional clinical testing […]
Catabasis has conceded defeat with its Duchenne muscular dystrophy drug edasalonexent, pulling the plug on the drug after a phase 3 trial echoed the results […]
Date: October 19, 2020 Issue #: 1609 Summary: The antisense oligonucleotide viltolarsen (Viltepso – NS Pharma) has received accelerated approval from the FDA for treatment […]
The FDA has approved a new therapy for the rare muscle wasting disease Duchenne muscular dystrophy (DMD) as Japan’s NS Pharma takes on Sarepta and […]
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