Cure CMD

January 2012 Congenital Muscle Disorder Newsletter: Scoliosis

Unab — Sat, 04 Feb 2012 03:59:15 +0000

Podcast: Stephen Tapscott Discusses Role of DUX4 in FSHD

admin — Fri, 03 Feb 2012 00:43:26 +0000

An 11½-minute interview with researcher Stephen Tapscott explores recent findings about the molecular basis of facioscapulohumeral MD

MDA Awards More Than $12 Million in Grants to Advance Neuromuscular Disease Research

admin — Thu, 02 Feb 2012 00:35:08 +0000

The Muscular Dystrophy Association's latest round of grants allocates more than $12 million to the support of 38 research projects investigating the causes of, and potential treatments for, a number of forms of neuromuscular disease.

Jacobus Begins Invitation-Only Trial of 3,4-DAP in LEMS

admin — Wed, 01 Feb 2012 20:34:50 +0000

Jacobus is inviting 30 selected individuals with Lambert-Eaton myasthenic syndrome to participate in a placebo-controlled trial of 3,4-DAP.

MDA Awards $2 Million in ALS Grants

admin — Wed, 01 Feb 2012 20:34:49 +0000

The grants fund six new ALS projects, each using a different mechanism to uncover the causes of the disease and potential treatments.

MDA Commits $12 Million to Neuromuscular Disease Research

admin — Wed, 01 Feb 2012 20:34:48 +0000

MDA has awarded 38 grants totaling more than $12 million to support research into general muscle health and more than 15 neuromuscular diseases in its program.

Cure CMD, LGMD2I Fund and TREAT-NMD/Newcastle: Teaming Up to Prepare for LGMD2I Trial

anne_rutkowski — Mon, 30 Jan 2012 06:41:04 +0000

Cure CMD, LGMD2I Fund and Newcastle office are teaming up to prepare for an LGMD2I clinical trial.

First step, harmonizing registries. If FKRP, please register in www.fkrp-registry.org If any other aDG-RD gene involved or unknown, please register in CMDIR, www.cmdir.org

FKRP and CMDIR Registries: Working Together

anne_rutkowski — Mon, 30 Jan 2012 06:38:52 +0000

Cure CMD, the LGMD2I Fund and the Newcastle TREAT-NMD office announce a collaboration to promote clinical trial readiness for LGMD2I. In anticipation of an LGMD2I clinical trial, we are working to align the 2 existing registries, one specific for FKRP and one for each of the other CMD-LGMD alpha dystroglycan related dystrophy (aDG-RD, dystroglycanopathy) genes. To launch a specific LGMD2I trial, we will need to have all FKRP patient information in one location, the FKRP international registry.

We would like to ask all people with confirmed FKRP mutations to register in the international FKRP registry (www.fkrp-registry.org). We would like to ask all people with a confirmed mutations in each of the other aDG related genes, including POMT1, POMT2, POMGnT1, DAG, LARGE and fukutin and those without a genetic diagnosis to register in the CMD International Registry (CMDIR-www.cmdir.org).

Both registries will continue to work together to provide registrants with the same up to date information on clinical studies and trials in the aDG-RDs. We believe that strides made in clinical trial readiness for the LGMD2I population will have significant impact for the larger group of patients with alpha-dystroglycan related dystrophies (aDG-RDs). We would like to thank all who have currently registered and encourage registrants to fully complete the survey questions.

January 2012 Cure CMD Newsletter

Unab — Wed, 25 Jan 2012 22:11:52 +0000

Click here to view the January 2012 Newsletter

Phase 1 Trial Opens of ISIS-SMNRx in Children with SMA

admin — Mon, 23 Jan 2012 22:58:56 +0000

Pharmaceuticals will test its experimental antisense drug in 24 children with spinal muscular atrophy at five U.S. centers.