AveXis Submits BLA for SMA Gene Therapy AVXS-101 to FDA

This week, AveXis, a Novartis company, reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of AVXS-101 to treat infants with spinal muscular atrophy (SMA) type 1. AveXis is also submitting regulatory applications for the therapy in Europe and Japan. If approved, AVXS-101 would . . .

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Pharnext Announces Positive Results from Phase 3 Trial Using PXT3003 to Treat CMT1A

Today, French pharmaceutical company Pharnext SA announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). PLEO-CMT was a pivotal, 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 compared . . .

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Sarepta Therapeutics Announces Positive Updated Results from Gene Therapy Trial for Duchenne Muscular Dystrophy

This week, at the 23rd International Congress of the World Muscle Society in Argentina, Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, shared additional data relating to Sarepta Therapeutics’ AAVrh74.MHCK7.micro-Dystrophin gene therapy program for Duchenne muscular dystrophy (DMD).  Dr. Mendell had previously presented results for the first three patients in the study back in . . .

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Researchers at SUNY Binghamton Seek Participants for BMD Biomarker and Patient-Reported Outcomes Study

Researchers at the State University of New York at Binghamton’s School of Pharmacy and Pharmaceutical Sciences are seeking participants living with Becker muscular dystrophy (BMD) to participate in a biomarker and patient-reported outcomes study. The purpose of the Becker Muscular Dystrophy Biomarker and Patient-Reported Outcomes Study is to learn more about disease progression in patients . . .

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Genentech Announces New Data for Risdiplam in SMA

On Oct. 3, Genentech announced interim results from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). Risdiplam is an investigational SMN2-splicing modifier that is given by mouth (or g-tube) and distributes widely throughout the body. Risdiplam is designed to help the SMN2 gene produce more . . .

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Clinical Trial Alert: Capricor Seeks Participants for a Phase 2 DMD Study

Researchers at Capricor Therapeutics are looking for ambulatory and non-ambulatory children and adults with Duchenne muscular dystrophy (DMD) to participate in a Phase 2 study. The goal of the study is to evaluate the safety and efficacy of CAP-1002. Researchers hope that this therapy may slow disease progression in heart and skeletal muscle by modulating . . .

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The Changing Landscape of LGMD Research: A Conversation with MDA Scientific Portfolio Director Lianna Orlando

Sept. 30th is Limb-Girdle Muscular Dystrophy Awareness Day, which celebrates individuals living with the more than 30 different subtypes of Limb-girdle muscular dystrophy (LGMD). MDA has a long-standing commitment to LGMD — we’ve invested more than $59 million in LGMD-specific research since 1950, including more than $2.8 million in current active grants. MDA Scientific Portfolio . . .

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UC Davis Researchers Seek DMD Participants for Survey to Improve Clinical Trial Outcome Measurements

In order to develop better methods for capturing the perspective of individuals living with Duchenne muscular dystrophy (DMD) during clinical trials, researchers at the University of California Davis Neuromuscular Research Center are seeking participants for an online survey to monitor movement and daily living abilities. The Duchenne Muscular Dystrophy Life-Time Mobility Scale (DMD-LMS) is a . . .

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FDA Announces the Approval of TIGLUTIK, an Oral Suspension Form of riluzole, for the Treatment of ALS

On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK, marketed by ITF Pharma, the U.S. subsidiary of Italian specialty pharmaceutical company Italfarmaco, is an oral suspension formulation of riluzole, which has been available in . . .

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Three Major Patient Advocacy Groups Focused on CMT Research to Collaborate on FDA “Voice of the Patient Report” and Patient-Focused Meeting

In honor of Charcot-Marie-Tooth (CMT) Awareness Month, the Muscular Dystrophy Association joins the Charcot-Marie-Tooth Association (CMTA) and the Hereditary Neuropathy Foundation (HNF) to host an externally led patient-focused drug development (PFDD) meeting for CMT. The PFDD meeting is being held on Friday, Sept. 28 at the College Park Marriott Hotel and Conference Center in Hyattsville, . . .

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