Category: Research

News, Research

Reata Pharmaceuticals Announces Positive Results from Pivotal Study of Omaveloxolone in FA

Jeanene Swanson

Texas-based Reata Pharmaceuticals announced positive results of its pivotal phase 2 clinical trial (MOXIe) of omaveloxolone in patients with Friedreich’s ataxia (FA). Patients treated with omaveloxolone showed a statistically significant improvement in the modified Friedreich’s Ataxia Rating Scale mFARS (a scale used to measure neurological function) after 48 weeks of treatment compared to placebo, and . . .

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News, Research

Capricor Therapeutics Announces Positive Interim Results from Phase 2 Study of CAP-1002 to Treat DMD

Jeanene Swanson

In July of this year, Capricor Therapeutics announced positive interim results from its phase 2 (HOPE-2) clinical trial evaluating CAP-1002, the company’s lead investigational therapy for Duchenne muscular dystrophy (DMD), to treat boys and young men in advanced stages of the disease. In that analysis, meaningful improvements in mid-level upper limb and hand function were . . .

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News, Research

MDA Grants Charles Thornton More Than $1 Million in New Funding to Continue Supporting the Myotonic Dystrophy Clinical Research Network

Jeanene Swanson

Charles Thornton, MD, professor of Neurology at the University of Rochester, was awarded an MDA clinical research network grant (CRNG) totaling $1,118,673 over three years to continue to lead the development of the Myotonic Dystrophy Clinical Research Network, which comprises six medical centers specializing in research and clinical care of myotonic dystrophy (DM) types 1 . . .

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News, Research

Five Questions with DMD Researcher Matthew Wood

Jeanene Swanson

Matthew Wood, PhD, professor of Neuroscience at the University of Oxford and director of both the Oxford Rare Disease Center and the MDUK Oxford Neuromuscular Center, was awarded an MDA research grant totaling $210,000 over two years to optimize the delivery of genetic therapies such as oligonucleotides to tissues for treating Duchenne muscular dystrophy (DMD). . . .

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News, Research

ReveraGen Presents Positive Interim Results from Phase 2 Extension Study of Vamorolone to Treat DMD in Young Boys

Jeanene Swanson

At the World Muscle Society annual meeting held earlier this month in Copenhagen, Denmark, ReveraGen announced positive results from its ongoing phase 2 clinical trial of vamorolone for treating Duchenne muscular dystrophy (DMD) in boys. Building upon previously reported positive results, CEO Dr. Eric Hoffman presented data gathered after 18 months of treatment in the . . .

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Personal Stories, Research

At a Cedars-Sinai Care Center, Dr. Robert Baloh Looks Under — and Ahead of — the Microscope for CMT and ALS

Lindsey Baker

Even though the Martinez family lives in Burbank, Calif., within an hour’s drive to Los Angeles’ Cedars-Sinai Medical Center, their road to the hospital’s specialized clinics — to Dr. Robert Baloh and his work there — was a long one. Daniel and Gladis Martinez have two daughters: Genesis, 15, and Isabella, 14, born just 10 . . .

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Research

Five Questions with LGMD Researcher Martin Childers

Jeanene Swanson

Martin Childers, PhD, DO, chief medical officer at Asklepios BioPharmaceutical Inc. in North Carolina, was awarded an MDA research grant totaling $192,500 over one year to perform pre-clinical studies using an adeno-associated virus (AAV) to deliver a gene therapy for limb-girdle muscular dystrophy type 2I (LGMD2I). Limb-girdle muscular dystrophies are a diverse group of disorders . . .

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News, Research

MDA Awards Clinical Research Network Grant to CMT Researcher Michael Shy for Inherited Neuropathies Consortium

Jeanene Swanson

Michael Shy, PhD, professor of Neurology and Pediatrics at the University of Iowa, was awarded an MDA Clinical Research Network Grant (CRNG) totaling $423,413 over three years to further develop the Inherited Neuropathies Consortium (INC), a network of clinical investigators dedicated to developing the infrastructure necessary to evaluate therapies for patients with inherited peripheral neuropathies, . . .

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News, Research

In a Letter to the Duchenne Community, Santhera Announces Positive Results of Phase 2 Vamorolone Study, EMA’s Review of Puldysa to Treat Symptoms of DMD

MDA Staff

This week, Santhera released a letter to the Duchenne muscular dystrophy (DMD) community regarding an update on Santhera’s DMD drug development programs and its partnership with ReveraGen. The full letter follows. Letter to the DMD community from Santhera: Dear Duchenne community, It was great to connect with so many of you at the Parent Project . . .

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