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Research - General

The Brain in MMD (September/October 2008)

Function of Children with Type 2 SMA Improved with Albuterol (September/October 2008)

Researchers ID Protein that Drastically Lessens Severity of SMA in Females (September/October 2008)

Study of 25 Found SBMA has Small Impact on Function, Survival (September/October 2008)

Brain MRI Abnormalities in MMD1 Similar Within Families (September/October 2008)

Specific Cardiac Problem Seen in MMD2 (September/October 2008)

Risk of Sudden Death 3 to 5 Times Above Average in Type 1 Myotonic Dystrophy (September/October 2008)

ID of Protein that Prevents Myelin Formation Could Have Implications for CMT Treatment (September/October 2008)

Mitochondrial Disease Meeting Highlights New Directions (September/October 2008)

Myotilin Protein may be a Viable Therapeutic Target in LGMD1A (September/October 2008)

Utrophin-Stimulating Compound Improves Strength, Endurance in Mice (September/October 2008)

Viagra Improved Heart Function in DMD Mice (September/October 2008)

New Directions: Can an Immune Response be Rerouted to Treat Disease? (July/August 2008)

MDA Supports 1st U.S. Trial of Lithium in ALS (July/August 2008)

Valproic Acid and Carnitine to be Tested in SMA1 (July/August 2008)

CMT1A May Worsen with Passage from Parent to Child (July/August 2008)

Four Small Studies Found Rituximab May Help in Treatment-Resistant MG (July/August 2008)

Antibodies to SOX1 Protein May Indicate Lung Cancer in LEMS (July/August 2008)

Boys with DMD or BMD Responded to ACE Inhibitor Treatment of Heart Problems (July/August 2008)

Dystrophin Mutation Analysis Predicted Age of Onset of Cardiac Problems in BMD (July/August 2008)

Pentoxifylline Benefits Minimal in DMD (July/August 2008)

Compressed Prednisone Schedule in DMD May Reduce Some Side Effects (July/August 2008)

MDA Joins SMA Groups in Support of SMA Treatment Acceleration Act (July/August 2008)

Company to Develop ‘Chaperon’ Molecule for Pompe Disease (July/August 2008)

Protecting Mitochondria with Debio-025 Helped Mice with Two Forms of MD (July/August 2008)

Gene Transfer Benefits Mice with Myotubular Myopathy (July/August 2008)

Gene Transfer May be a Good Way to Block Myostatin (July/August 2008)

A Little Dystrophin is Better Than None (July/August 2008)

Gene ID Translates Into Test for CNM and CMT (May/June 2008)

Caregiving Parents to be Surveyed (May/June 2008)

Study to Evaluate Removing Thymus in MG Remains Open (May/June 2008)

Phase 3 Trial of Idebenone in Friedreich’s Ataxia is Open (May/June 2008)

Daily Versus Weekly Prednisone Study in DMD is Now Complete (May/June 2008)

Yearlong Study of PTC124 in DMD, BMD is Open (May/June 2008)

Sodium Phenylbutyrate Being Tested in Types 1, 2 and 3 SMA and in Presymptomatic Infants (May/June 2008)

Trial of Vitamin C in CMT1A Still Open (May/June 2008)

Iplex is in Phase 2 Trial for MMD1 (May/June 2008)

Screening for Gene Transfer Study in LGMD2D Continues (May/June 2008)

Wyeth Won’t Continue Developing MYO-029 (May/June 2008)

MDA and FARA Team up to Support Research in Friedreich’s Ataxia (May/June 2008)

Nerve-Muscle Signals go Both Ways (May/June 2008)

Three New Studies Shed Light on Myostatin Blocking to Treat MD (May/June 2008)

Progress in SMA Research (May/June 2008)

Flaws in FHL1 Gene Implicated in Rare Myopathies (May/June 2008)

New Type of MMD Mouse Adds to Understanding of Human Disease (May/June 2008)

More DMD, BMD Patients May Benefit From Exon Skipping (May/June 2008)

Not Enough ZZZzzzs? (March/April 2008)

MDA and Chest Physicians Release Recommendations for Anesthesia Care in DMD (March/April 2008)

Myozyme Improves Endurance, Pulmonary Function in Late-onset Pompe Disease (March/April 2008)

Children with Severe SMA are Surviving Longer (March/April 2008)

Amarin will Continue Testing EN101 in MG (March/April 2008)

Gene Transfer Appears Safe in Six Boys with DMD (March/April 2008)

PTC Therapeutics to Start Longer Studies of PTC124 (March/April 2008)

Results of Phase 1 Trial of Exon Skipping in DMD Heralded as ‘Proof of Concept’ (March/April 2008)

New Studies Improve Understanding of Myotonic Dystrophy (March/April 2008)

MDA Grantee Receives Prestigious Pfizer Award (March/April 2008)

Gene ID’d for X-linked SMA (March/April 2008)

Embryonic Stem Cells Benefit Muscles in DMD-affected Mice (March/April 2008)

European Team Combines Stem Cells, Gene Correction to Treat DMD-affected Mice (March/April 2008)

Adding to the Mix – New Strategies are Positioning MDA’s Research Program for Success (Jan./Feb. 2008)

Moving Away From the Blasts of the Past (Jan./Feb. 2008)

Brain Involvement Appears Less Serious in MMD2 Than MMD1 (Jan./Feb. 2008)

Santhera Drug Also May Help in Friedreich’s Ataxia (Jan./Feb. 2008)

Santhera Drug May Help DMD-affected Hearts, Respiratory Muscles (Jan./Feb. 2008)

Trial of Exon Skipping in DMD Opens in UK (Jan./Feb. 2008)

PTC124 Allowed Dystrophin Production in Half of DMD-affected Trial Participants (Jan./Feb. 2008)

Bone Development in DMD Under Study (Jan./Feb. 2008)

SMA Groups Hear Biotech Presentations, Plan Trials (Jan./Feb. 2008)

TREAT-NMD Goes Global (Jan./Feb. 2008)

HDAC Inhibitors Quiet Immune Response by Boosting Regulatory Cells (Jan./Feb. 2008)

Myocardin Represses Skeletal Muscle, Promotes Smooth-muscle Development (Jan./Feb. 2008)

Suppressing MyoD Could Help Immature Muscle Cells Survive Transplantation (Jan./Feb. 2008)

T-cells in IBM Suggest Immune Response Against Muscle in Part of Disease (Jan./Feb. 2008)

MicroRNA Molecules May Provide New Therapeutic Targets in Several Diseases (Jan./Feb. 2008)

Blocking Calpain Helpful in Mice with Slow-channel Myasthenic Syndrome (Jan./Feb. 2008)

Dystrophin in 50 Percent of Heart Cells Protects DMD-affected Mice (Jan./Feb. 2008)

Penn Researchers Want to Break a Vicious Cycle (Jan./Feb. 2008)

What Not to Eat – Diet in 3 Metabolic Diseases (Nov./Dec. 2007)

Debatable Destinies – Duchenne Muscular Dystrophy Carriers Carry on, Despite Uncertainty (Nov./Dec. 2007)

Dysferlin Deficiency can Cause Heart Damage (Nov./Dec. 2007)

Anti-cell-death Compound to be Tested in CMD (Nov./Dec. 2007)

Boston Group Will Study Molecular Pathways in Several Muscle Diseases (Nov./Dec. 2007)

New Myozyme Studies to Provide More Data on Pompe Disease Treatment (Nov./Dec. 2007)

Two Meds for Periodic Paralysis Will be Tested (Nov./Dec. 2007)

Small Study Supports Development of Antisense Molecule for MG (Nov./Dec. 2007)

10-year Study Suggests Early ACE Inhibitor Treatment in DMD May Prolong Survival (Nov./Dec. 2007)

Saving Muscle May Require Moving Beyond Myostatin Blocking (Nov./Dec. 2007)

Decorin Protein Pushes Damaged Muscle Toward Repair (Nov./Dec. 2007)

New MMD1 Studies Connect CUG Binding Protein Overload to Errors in Molecular Splicing (Nov./Dec. 2007)

Excess of Mu-crystallin Protein Added to Hypotheses About FSHD (Nov./Dec. 2007)

SMA Standard of Care Committee Releases Disease Management Guidelines (Nov./Dec. 2007)

Utrophin Gene Therapy Benefits Mice With DMD (Nov./Dec. 2007)

A Range of Outcomes – Myotubular Myopathy (Sept./Oct. 2007)

Longer Survival in DMD With Ventilation, Scoliosis Surgery (Sept./Oct. 2007)

Steroid-Treated Boys Walk Longer, Have More Leg and Vertebral Fractures (Sept./Oct. 2007)

Insmed Planning Additional Studies of Iplex in Myotonic Dystrophy (Sept./Oct. 2007)

New Myozyme Studies to Provide More Data on Pompe’s Treatment (Sept./Oct. 2007)

Researchers Will Test High-Dose Vitamin C in CMT1A (Sept./Oct. 2007)

Z-Shaped Construct has Potential for Blocking Hazardous Genes (Sept./Oct. 2007)

Delivering Genes Via Bacteria May Become an Option (Sept./Oct. 2007)

Gene Transfer Using Herpes Virus Transporter Helps Mice With Friedreich's Ataxia (Sept./Oct. 2007)

Research Points to New Directions in Inclusion-Body Myositis Treatment (Sept./Oct. 2007)

ID of Utrophin Brake Raises DMD Treatment Hopes (Sept./Oct. 2007)

Do You Want to Know? – Prenatal & Newborn Genetic Diagnosis (July/August 2007)

Tackling DMD on Many Fronts (July/August 2007)

Experimental Compound Shows Promise for SBMA
(July/August 2007)

Female Muscle Stem Cells Work Better (July/August 2007)

Clinical Trials and Studies (July/August 2007)

History of Corticosteroids (May/June 2007, Quest Extra)

New Muscle Generating Stem Cells Isolated From Human Tissue (May/June 2007)

Two Anti-Fibrosis Drug Show Promise in Mice with DMD (May/June 2007)

Cyclosporine-Like Compound Prevents Cell Death in Mice with CMD (May/June 2007)

What Does Blocking Myostatin Do? (May/June 2007)

SMA Researchers Break New Ground (May/June 2007)

Not Always Smooth Sailing — Steroids & NMD (May/June 2007)

Impossible Things (March/April 2007)

Stem Cells Treat MD in Dogs (Jan./Feb. 2007)

Two Stem Cell Types May Be Better Than One (Jan./Feb. 2007)

Gene Therapy Now in Its Teen Years (Jan./Feb. 2007)

To Register or Not to Register: Research Registries (Nov./Dec. 2006)
FARA Launches Friedreich's Registry (Sept. 2006)

Merg1a Protein Could Be New Target in Fighting Atrophy (Sept. 2006)

Secrets of the Salamander (Nov. 2005)

Changing the Code: Can a Faulty Gene Be Saved? (Mar./Apr. 2005)

Bridge Over Troubled Waters (gene therapy) Jan./Feb. 2005)

Eggs in Many Baskets (Jeffrey Chamberlain, DMD researcher)

Out of the Ivory Tower (R. Jude Samulski, DMD researcher)

Delivering DNA Without Viruses (Jon Wolff, DMD researcher)

A Perfect Combination (DMD researcher Xiao Xiao)

Stem Cells With Muscle Potential Found (Nov./Dec. 2004)

When the Media Cover Medicine (Mar./Apr. 2004)

Putting Out the Fire (myositis) (Jan./Feb. 2004)

Understanding the Beast Within (autoimmune diseases) (Nov./Dec. 2003)

New Stem Cells Move From Blood to Muscle, Slow MD in Mice (Sept./Oct. 2003)

Could Proteins Activate Muscle Stem Cells? (Sept./Oct. 2003)

When Cells Kill Themselves (July/Aug. 2003)

Pompe's Disease: A Killer Yields to Modern Medicine (Mar./Apr. 2003)

From Steroids to Stem Cells (Feb. 2003)

Good News, Bad News on Stem Cells for Duchenne MD (Oct. 2002)

Kennedy's Disease (June 2002)

Transplant Study Reveals Heart's Regenerative Powers (Apr. 2002)

Family Histories Help Solve Medical Mysteries (Feb. 2002)

Stem Cell Therapy the Huard Way (Feb. 2002)

Myotonia Congenita (Dec. 2001)

Clinical Trials (Oct. 2001)

Fast-Track Pharmacy (Aug. 2001)

Neuromuscular Terminology (Aug. 2001)

Opening Up the Ivory Tower (Apr. 2001)

CMT and DS: From Clear-Cut Endings to Complex Beginnings (Feb. 2001)

Germ Line Mosaicism (Feb. 2001)

Stem Cell "Switch" Possible Key to Therapy (Feb. 2001)

Renewing Muscles and Nerves (Apr. 2000)

Gene Therapy Goes Like Clockwork (Oct. 1999)

Carnitine and Coenzyme Q10: Miracle Cures or Money Down the Drain? (Feb. 1999)

But Girls Don't Get Duchenne (Dec. 1998)

Hooked on Mitochondria (Oct. 1998)

Spotlight on Gene Therapy and Muscle (Aug. 1998)

The Immune System: The Body at War With a Cure (Aug. 1997)

Autoimmune Diseases: The Body at War With Itself (June 1997)

APPROVED! — Ushering Rilutek from the laboratory...through clinical trials...government approval...and into your hands (Winter 1996)

MDA Research Program

Recent Advances in DMD Gene Therapy (September/October 2008)

Risk of Sudden Death 3 to 5 Times Above Average in Type 1 Myotonic Dystrophy (September/October 2008)

Mitochondrial Disease Meeting Highlights New Directions (September/October 2008)

Myotilin Protein may be a Viable Therapeutic Target in LGMD1A (September/October 2008)

Viagra Improved Heart Function in DMD Mice (September/October 2008)

Brothers’ FSHD Spurs His Research (July/August 2008)

New Directions: Can an Immune Response be Rerouted to Treat Disease? (July/August 2008)

MDA Supports 1st U.S. Trial of Lithium in ALS (July/August 2008)

Four Small Studies Found Rituximab May Help in Treatment-Resistant MG (July/August 2008)

Boys with DMD or BMD Responded to ACE Inhibitor Treatment of Heart Problems (July/August 2008)

Dystrophin Mutation Analysis Predicted Age of Onset of Cardiac Problems in BMD (July/August 2008)

Compressed Prednisone Schedule in DMD May Reduce Some Side Effects (July/August 2008)

MDA Joins SMA Groups in Support of SMA Treatment Acceleration Act (July/August 2008)

A Little Dystrophin is Better Than None (July/August 2008)

Gene ID Translates into Test for CNM and CMT (May/June 2008)

Yearlong Study of PTC124 in DMD, BMD is Open (May/June 2008)

Screening for Gene Transfer Study in LGMD2D Continues (May/June 2008)

Wyeth Won’t Continue Developing MYO-029 (May/June 2008)

MDA and FARA Team up to Support Research in Friedreich’s Ataxia (May/June 2008)

Nerve-Muscle Signals go Both Ways (May/June 2008)

Three New Studies Shed Light on Myostatin Blocking to Treat MD (May/June 2008)

Flaws in FHL1 Gene Implicated in Rare Myopathies (May/June 2008)

More DMD, BMD Patients May Benefit From Exon Skipping (May/June 2008)

MDA and Chest Physicians Release Recommendations for Anesthesia Care in DMD (March/April 2008)

Myozyme Improves Endurance, Pulmonary Function in Late-onset Pompe Disease (March/April 2008)

Children with Severe SMA are Surviving Longer (March/April 2008)

Gene Transfer Appears Safe in Six Boys with DMD (March/April 2008)

PTC Therapeutics to Start Longer Studies of PTC124 (March/April 2008)

Results of Phase 1 Trial of Exon Skipping in DMD Heralded as ‘Proof of Concept’ (March/April 2008)

Results of Phase 1 Trial of Exon Skipping in DMD Heralded as ‘Proof of Concept’ (March/April 2008)

New Studies Improve Understanding of Myotonic Dystrophy (March/April 2008)

MDA Grantee Receives Prestigious Pfizer Award (March/April 2008)

Gene ID’d for X-linked SMA (March/April 2008)

European Team Combines Stem Cells, Gene Correction to Treat DMD-affected Mice (March/April 2008)

Adding to the Mix – New Strategies are Positioning MDA’s Research Program for Success (Jan./Feb. 2008)

Moving Away From the Blasts of the Past (Jan./Feb. 2008)

Santhera Drug Also May Help in Friedreich’s Ataxia (Jan./Feb. 2008)

PTC124 Allowed Dystrophin Production in Half of DMD-affected Trial Participants (Jan./Feb. 2008)

Bone Development in DMD Under Study (Jan./Feb. 2008)

SMA Groups Hear Biotech Presentations, Plan Trials (Jan./Feb. 2008)

TREAT-NMD Goes Global (Jan./Feb. 2008)

Suppressing MyoD Could Help Immature Muscle Cells Survive Transplantation (Jan./Feb. 2008)

MicroRNA Molecules May Provide New Therapeutic Targets in Several Diseases (Jan./Feb. 2008)

Blocking Calpain Helpful in Mice with Slow-channel Myasthenic Syndrome (Jan./Feb. 2008)

Dystrophin in 50 Percent of Heart Cells Protects DMD-affected Mice (Jan./Feb. 2008)

Penn Researchers Want to Break a Vicious Cycle (Jan./Feb. 2008)

Debatable Destinies – Duchenne Muscular Dystrophy Carriers Carry on, Despite Uncertainty (Nov./Dec. 2007)

Dysferlin Deficiency can Cause Heart Damage (Nov./Dec. 2007)

Boston Group Will Study Molecular Pathways in Several Muscle Diseases (Nov./Dec. 2007)

New Myozyme Studies to Provide More Data on Pompe Disease Treatment (Nov./Dec. 2007)

Two Meds for Periodic Paralysis Will be Tested (Nov./Dec. 2007)

Saving Muscle May Require Moving Beyond Myostatin Blocking (Nov./Dec. 2007)

New MMD1 Studies Connect CUG Binding Protein Overload to Errors in Molecular Splicing (Nov./Dec. 2007)

Excess of Mu-crystallin Protein Added to Hypotheses About FSHD (Nov./Dec. 2007)

Utrophin Gene Therapy Benefits Mice With DMD (Nov./Dec. 2007)

A Range of Outcomes – Myotubular Myopathy (Sept./Oct. 2007)

Steroid-Treated Boys Walk Longer, Have More Leg and Vertebral Fractures (Sept./Oct. 2007)

New Myozyme Studies to Provide More Data on Pompe’s Treatment (Sept./Oct. 2007)

Researchers Will Test High-Dose Vitamin C in CMT1A (Sept./Oct. 2007)

Research Points to New Directions in Inclusion-Body Myositis Treatment (Sept./Oct. 2007)

ID of Utrophin Brake Raises DMD Treatment Hopes (Sept./Oct. 2007)

Do You Want to Know? – Prenatal & Newborn Genetic Diagnosis (July/August 2007)

Tackling DMD on Many Fronts (July/August 2007)

Experimental Compound Shows Promise for SBMA
(July/August 2007)

Female Muscle Stem Cells Work Better (July/August 2007)

Not Always Smooth Sailing — Steroids & NMD (May/June 2007)

History of Corticosteroids (May/June 2007, Quest Extra)

Coping with Corticosteroids (May/June 2007, Quest Extra)

Nutritional Considerations (May/June 2007, Quest Extra)

Impossible Things (March/April 2007)

Defensive Action (Jan./Feb. 2007)

Two Lines of Myotonic Dystrophy Research Bear Fruit (Nov./Dec. 2006)
LGMD Gene Transfer Trial Slated for Next Year (Nov./Dec. 2006)
Androgen Receptors, Protein Blocking Explored in SBMA (Sept. 2006)

Stem Cells Restore Movement in Rats: May Apply in SMA, ALS (Sept. 2006)

Scientists Share Gene Therapy Advances (Sept. 2006)

Exon Skipping Skips Ahead (Sept. 2006)

CellCept Has Risks in Dermatomyositis (July 2006)

Statin Treatment May Disclose Metabolic Disorder (July 2006)

Closer Look Questions Risks of Medications for People With CMT (July 2006)

MMD Research (July 2006)

SMA Studies (July 2006)

Three Labs Find Microdystrophin Genes Effective (July 2006)

Arterial Delivery Promising from Muscle Stem Cells (July 2006)

FDA Approves Myozyme for Pompe's Disease (July 2006)

CoQ10 Deficiency Rare and Easily Treatable (May 2006)

In MELAS, Side Effects of DCA Outweigh Benefits (May 2006)

Etanercept Study in IBM Somewhat Encouraging (May 2006)

Membrane Damage Added to FSHD Picture (May 2006)

More Genes Implicated in Charcot-Marie-Tooth (May 2006)

Mouse Studies Bode Well for Exon Skipping in DMD (May 2006)

MDA Begins First U.S. Duchenne Gene Therapy Trial (May 2006)

Mending the Membrane, Priming the Pump: Foreseeing the Future of Cardiac Care in MD (May 2006)

Results Negative for Diltiazem in FSHD (March 2006)

MMD2 Rarity Questioned (March 2006)

Altered Protein Related to Autoimmune Diseases (March 2006)

New Myostatin Blocker Enlarges Mouse Muscles (March 2006)

Protein Flaw Turns on Heart-Damaging Genes in 2 Dystrophies (March 2006)

Injecting Second Gene in Mice Enhances Dystrophin Gene Therapy (March 2006)

Team Ids Consequences of FSH MD Mutation (March 2006)

MDA Supports Bill to Ease Biotech Restrictions (Jan. 2006)

MDA Grantee Receives NIH Pioneer Award (Jan. 2006)

NIH Names Three New MD Centers of Excellence (Jan. 2006)

Mexiletine Safe, Helpful for MMD-Related Myotonia (Jan. 2006)

Sleepiness in MMD1 Not Due to Low Orexin (Jan. 2006)

Preclinical Studies Show Progress in MD Gene Therapy (Jan. 2006)

Protein May Increase Frataxin in Friedreich's Ataxia (Jan. 2006)

Heart Damage Varies With Dystophin Mutations (Jan. 2006)

Early Diagnosis, Treatment Improve Cardiac Outloook in Duchenn, Becker MDs (Jan. 2006)

Connecting the Dots: Research Shows Congenital MDs Have Roots Outside Cells (Jan. 2006)

MDA Gathers Scientists, Physicians for Updates on Research Progress (Jan. 2006)

Blocking Immune System's IL1 Helps Mice With MG (Nov. 2005)

New Compound Protects Hearts From DMD Damage (Nov. 2005)

SMA Cells Get Boost From Hydroxyurea (Nov. 2005)

Two Genes Better Than One in Mice With Duchenne MD (Nov. 2005)

Modifier Genes Change Severity of Limb-Girdle MD (Nov. 2005)

Compounds Up Utrophin, Help DMD-Affected Mice (Nov. 2005)

Genes for Agrin Treat Mice Lacking Laminin (Nov. 2005)

Secrets of the Salamander (Nov. 2005)

Grantees Honored (Sept. 2005)

Researchers Explore Muscle Stem Cells (Sept. 2005)

University of Iowa Named Wellstone Center (Sept. 2005)

Tipping the Balance: Can Scientists Build Muscle Tissue Safely? (Sept. 2005)

Cardiac Stem Cells Come Closer to Clinic (May 2005)

NIH/MDA Centers of Excellence (Sept./Oct. 2004)

MDA, Others Explore Value of Steroid Use in DMD (July/Aug. 2004)

ENMC Report

Cochrane Review

New Hope in SMA (May/June 2004)

MDA Grantee Receives Prestigious Award (Jan./Feb. 2004)

From Steroids to Stem Cells (Feb. 2003)

Halted LGMD Trial of Gene Therapy Shows No Benefit, No Harm (June 2002)

First Fruits of MD-CARE Act Lead to Meeting on Data Gathering (June 2002)

MDA Stem Cell Workshop Sets Research Plan (Apr. 2002)

MDA's Gene Therapy Program Back on Track (Apr. 2002)

MDA Suppports New Research Law (June 2001)

Opening Up the Ivory Tower (Apr. 2001)

MDA Research At Year 2000 (Feb. 2000)

Gene Therapy Goes Like Clockwork (Oct. 1999)

MDA Research for the New Millennium (Oct. 1999)

Special Report: Gene Therapy Entering the Clinic (Aug. 1999)

MDA Researchers Gear Up for Clinical Trials in Gene Therapy (Oct. 1998)

Base Camp Reached by Gene Therapy Researchers (Oct. 1998)

Spotlight on Gene Therapy and Muscle (Aug. 1998)

Gene Therapy Clinical Trials Scheduled (Feb. 1998)

Gene Therapy Hovers on the Horizon (Aug. 1997)

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