Duchenne muscular dystrophy

Buoyed by new data, Santhera preps filing for DMD drug vamorolone

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 to file replacement drug vamorolone, armed with new data from a pivotal trial, in what looks like a reversal of its fortunes. With prospects for idebenone looking shaky, Santhera snapped …

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Capricor preps phase 3 for Duchenne cell therapy as it seeks a partner

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 showed a strong effect on disease progression. Initial results from the 20-patient HOPE-2 trial reported last year showed signs of activity in older boys and young men with advanced DMD, …

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Capricor’s muscle drug data look good, but partner wanted and Phase 3 test needed

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 3 test is needed to support a regulatory submission, and the biotech is hunting for a pharmaceutical industry partner.

Duchenne Muscular Dystrophy Market: What’s More Beyond Exon-Skipping Therapies?

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 fail. Symptoms often show up by the age of 5; as the disease progresses, patients tend to lose the ability to walk around the age of 12. As per a recent …

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Casimersen (Amondys 45) for Duchenne Muscular Dystrophy (online only)

Date:  June 28, 2021 Issue #:  1627 Summary:  Casimersen (Amondys 45 – Sarepta), an IV antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 45 skipping (DMD-45), which occur in ~8% of DMD cases. …

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Santhera and ReveraGen Report Results of Vamorolone in P-IIb VISION-DMD Study for Duchenne Muscular Dystrophy

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 aged 4 to <7 years with DMD for 48 wks. The study met its 1EP i.e improvement in TTSTAND velocity, following the 1st period @24 wks. The 2 EPs include …

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Sarepta’s SRP-9001 Demonstrates Robust Expression and Safety Results in ENDEAVOR Study for DMD

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 demonstrated robust transduction, with a mean micro-dystrophin expression of 55.4% of normal, as measured by western blot. Micro-dystrophin was properly localized to the muscle sarcolemma, with patients achieving a mean …

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First UK patient joins Pfizer’s phase 3 DMD gene therapy trial

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 US in January this year and has now begun enrolling eligible patients in the UK. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one …

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Scotland opens door to Translarna for Duchenne muscular dystrophy

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 PTC Therapeutics’ Translarna for the first time.  The Scottish Medicines Consortium (SMC) says that Translarna (ataluren) can be provided by NHS Scotland for a period of three years, once PTC …

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Catabasis craters after giving up on muscular dystrophy lead

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 of a failed mid-stage study. The PolarisDMD study failed across the board, leaving Catabasis with no choice but to abandon the drug, including an ongoing open-label extension study from an …

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Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy

Date:  October 19, 2020 Issue #:  1609 Summary:  The antisense oligonucleotide viltolarsen (Viltepso – NS Pharma) has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping (DMD-53). It is the second drug to be approved …

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NS Pharma takes on Sarepta as FDA approves DMD drug

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 its controversially approved rival. NS Pharma’s Viltepso (viltolarsen) has been approved in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The DMD …

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