Skip to content Skip to footer
Subscribe Now
NEWS

Santhera Pharmaceuticals Reports Health Canada’s Approval of Agamree (Vamorolone) to Treat Duchenne Muscular Dystrophy

Shots:Health Canada has approved Agamree for the treatment of pts (≥4yrs) with Duchenne muscular dystrophy under priority reviewApproval was based on VISION-DMD study, which met its 1EP of Time to Stand (TTSTAND) velocity vs PBO at 24wks. of treatment, plus showed favorable safety & tolerability in ptsKye Pharma secured exclusive Canadian rights…
October 6, 2025

Read more

NEWS

Solid Biosciences & Kinea Bio Partner to Advance KNA-155 Using AAV-SLB101 for Dysferlin-Related Limb-Girdle Muscular Dystrophy

Shots:Solid Biosciences has entered into a non-exclusive global license & collaboration agreement with Kinea Bio to develop & commercialize KNA-155 for treating dysferlin-related limb-girdle muscular dystrophy, using Solid’s AAV-SLB101 capsidAs per the deal, Kinea Bio will receive a non-exclusive global license to utilize AAV-SLB101 for the delivery of KNA-155, which will enter IND-enabling studies…
September 24, 2025

Read more

Sarepta Therapeutics
NEWS

Sarepta Therapeutics Reports the MHLW’s Conditional & Time-Limited Approval of Elevidys for Duchenne Muscular Dystrophy (DMD)

Shots:Japan's MHLW has granted conditional & time-limited approval to Elevidys for DMD pts (3–8yrs.) with no exon 8/9 deletions in the DMD gene & who are negative for anti-AAVrh74 AbsApproval was based on extensive clinical data, incl. 2yr. global P-III (EMBARK) trial showing motor function benefits vs external controls; Part 2 data was…
May 14, 2025

Read more

Italfarmaco
NEWS

Italfarmaco Obtains the CHMP’s Positive Opinion for Conditional Marketing of Duvyzat to Treat Duchenne Muscular Dystrophy (DMD)

Shots:Opinion for Duvyzat to treat ambulant DMD pts (≥6yrs.) with corticosteroids is based on P-III (EPIDYS) study assessing Duvyzat (BID) vs PBO in ambulant boys (n=179); EC’s decision expected in Jul 2025Trial met its 1EP by improving four-stair climb completion time, with benefits seen in 2EPs  (NSAA & MRI fat infiltration) & a…
April 28, 2025

Read more

NEWS

Cumberland Pharmaceuticals Reveals Topline Data from P-II (FIGHT DMD) Trial of Ifetroban for Duchenne Muscular Dystrophy Heart Disease

Shots:Cumberland reported topline data from P-II (FIGHT DMD) trial assessing ifetroban (150/300mg, QD) vs PBO in DMD pts (n=41) for 12mos.; funded via the US FDA orphan products grant Study showed that ifetroban (300mg) led to 3.3% overall LVEF improvement (1EP), with a 1.8% increase vs 1.5% decline; on comparison with matched natural…
February 5, 2025

Read more

Viewpoints_Phil Lambert
VIEWPOINTS

Enhancing DMD Care: Phil Lambert from Satellos in a Riveting Conversation with PharmaShots

Shots: Conventional therapeutic models for Duchenne Muscular Dystrophy (DMD) primarily focus on the role of dystrophin in maintaining muscle cell integrity. However, Satellos’ SAT-3247 takes a novel approach, targeting the lack of dystrophin in muscle stem cells to restore muscle fiber regeneration and repair Today at PharmaShots, we are joined by Phil Lambert, Chief Scientific…
November 20, 2024

Read more

Italfarmaco
NEWS

Italfarmaco’s Duvyzat Obtains the US FDA’s Approval to Treat Duchenne Muscular Dystrophy 

Shots: The US FDA has granted approval to Italfarmaco’s Duvyzat (givinostat), histone deacetylase (HDAC) inhibitor, to treat Duchenne muscular dystrophy (DMD) patients (6yrs. or above) The approval was supported by the P-III (EPIDYS) study assessing the safety & efficacy of Duvyzat (BID) vs PBO in DMD ambulant male patients (n=179, 6yrs. or above)   The trial…
March 22, 2024

Read more

Load more