Muscular Dystrophy News Forums | MD forums Moderator | Activity https://musculardystrophynews.com/forums/members/md-forums-moderator/activity/ Activity feed for MD forums Moderator. Thu, 09 Apr 2026 07:35:58 -0500 https://buddypress.org/?v=2.20.0 en-US 30 hourly 2 fc797426ebd64472795ce89557096b4e MD forums Moderator started the discussion Collaboration Provides Cough Assist Machines to Neuromuscular Disease Patients in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/collaboration-provides-cough-assist-machines-to-neuromuscular-disease-patients/ Fri, 26 Jun 2020 15:11:49 -0500 Collaboration Provides Cough Assist Machines to Neuromuscular Disease Patients

A collaboration between Muscular Dystrophy Canada and Technology for Living seeks to provide cough assist machines to British Columbia residents. Read more about the partnership here.

What do you think of this news?

]]> 0ee0db6e8650dfe1598573c8c7c68211 MD forums Moderator started the discussion Sarepta's Gene Therapy for DMD Showing to Safety, Efficacy at One Year in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/sareptas-gene-therapy-for-dmd-showing-to-safety-efficacy-at-one-year/ Wed, 24 Jun 2020 14:56:29 -0500 Sarepta's Gene Therapy for DMD Showing to Safety, Efficacy at One Year

Four Duchenne boys given SRP-9001, a micro-dystrophin gene therapy, continue to show better muscle strength and safety at one year in a Phase 1/2 trial. Read more about their results here.

What do you think of this news?

]]> 8d49cc48e28d3bd3f1368ddfd7971619 MD forums Moderator started the discussion MDA Holds Q&A on Navigating COVID-19, and Area Reopenings, Safely in the forum COVID-19 and MD https://musculardystrophynews.com/forums/forums/topic/mda-holds-qa-on-navigating-covid-19-and-area-reopenings-safely/ Mon, 22 Jun 2020 15:54:37 -0500 MDA Holds Q&A on Navigating COVID-19, and Area Reopenings, Safely

Recently, the MDA held a Facebook Q&A with John Day, a Stanford University neurologist, to address the community’s concerns about the pandemic and personal safety. Click here to learn more about it.

What did you think of this Q&A?

]]> e8bab8bd9b50c79017f280bf8ca27df5 MD forums Moderator started the discussion Coalition Will Address Racial Disparities in Rare Disease Communities in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/coalition-will-address-racial-disparities-in-rare-disease-communities/ Wed, 17 Jun 2020 15:12:26 -0500 Coalition Will Address Racial Disparities in Rare Disease Communities

The Black Women’s Health Imperative has created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Read more about it here.

What are some specific topics or issues you hope this coalition will address?

]]> 5c248d0165b4f754a0154482a5f2a577 MD forums Moderator started the discussion Viltolarsen Earns European Orphan Drug Status as Duchenne Treatment in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/viltolarsen-earns-european-orphan-drug-status-as-duchenne-treatment/ Mon, 15 Jun 2020 14:02:23 -0500 Viltolarsen Earns European Orphan Drug Status as Duchenne Treatment

The European Commission has granted orphan drug designation to viltolarsen, a therapy for Duchenne muscular dystrophy patients amenable to exon 53 skipping. Click here to read more.

What do you think of this news?

]]> 54d253cd9132e71eb1ef0c19c4db2d93 MD forums Moderator started the discussion DMD Gene Therapy Research to Get $1M From PPMD, Duchenne UK in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/dmd-gene-therapy-research-to-get-1m-from-ppmd-duchenne-uk/ Fri, 12 Jun 2020 17:02:22 -0500 DMD Gene Therapy Research to Get $1M From PPMD, Duchenne UK

PPMD and Duchenne UK have announced a collaboration to provide up to $1 million in funding for gene therapy research in Duchenne muscular dystrophy. Read the full story here.

What do you think of this news?

]]> ca73509d0e6edd4df57ad0476444a43c MD forums Moderator started the discussion Santhera, Rutgers Partner on LAMA2 Muscular Dystrophy Gene Therapy in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/santhera-rutgers-partner-on-lama2-muscular-dystrophy-gene-therapy/ Wed, 10 Jun 2020 15:11:47 -0500 Santhera, Rutgers Partner on LAMA2 Muscular Dystrophy Gene Therapy

Santhera has announced agreements with Rutgers University to develop potential gene therapies for congenital MD type 1A focused on the use of linker proteins. Read more here.

What do you think of this news?

]]> 40ac8e2e221334a1ec4a074f4fa989fd MD forums Moderator started the discussion Why Self-monitoring Our Functioning Levels Is Important in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/why-self-monitoring-our-functioning-levels-is-important/ Mon, 08 Jun 2020 14:41:57 -0500 Why Self-monitoring Our Functioning Levels Is Important

Ralph Yaniz weighs in on the scientific progress in muscular dystrophy research since he was diagnosed with LGMD2L in 2006. Read more from Ralph here.

How much has science progressed since you were diagnosed with MD?

]]> da36be0825bffb9971a74065466d9891 MD forums Moderator started the discussion SIDEROS Trial of Puldysa in Duchenne Patients Completes Enrollment in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/sideros-trial-of-puldysa-in-duchenne-patients-completes-enrollment/ Thu, 04 Jun 2020 14:12:09 -0500 SIDEROS Trial of Puldysa in Duchenne Patients Completes Enrollment

Santhera said a next step for its Phase 3 trial of Puldysa in aiding lung function in DMD patients on glucocorticoids is independent analysis of efficacy. Click here to read more.

What do you think of this news?

]]> a3a300e767b3af27667a81c8ae4dc5cc MD forums Moderator started the discussion DMD Trial Supports ATL1102's Safety in Improving Muscle Strength in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/dmd-trial-supports-atl1102s-safety-in-improving-muscle-strength/ Mon, 01 Jun 2020 14:53:39 -0500 DMD Trial Supports ATL1102's Safety in Improving Muscle Strength

A Phase 2 clinical trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability in boys with Duchenne muscular dystrophy. Read more about it here.

What do you think of this news?

]]> 5705457c7811a73225b6d74e7752eb71 MD forums Moderator started the discussion Pfizer's DMD Gene Therapy Shows Promise in Improving Motor Abilities in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/pfizers-dmd-gene-therapy-shows-promise-in-improving-motor-abilities/ Fri, 29 May 2020 14:03:38 -0500 Pfizer's DMD Gene Therapy Shows Promise in Improving Motor Abilities

Updated trial data show improved motor abilities in boys with Duchenne muscular dystrophy who were given Pfizer’s experimental gene therapy PF-06939926. Read more here.

What do you think of this news?

]]> 1e9606187222a8800bcf98ac40cf0589 MD forums Moderator started the discussion Video Games Connect Chronically Ill Children Isolated at Home, Hospital in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/video-games-connect-chronically-ill-children-isolated-at-home-hospital/ Thu, 28 May 2020 14:02:25 -0500 Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Hospitals are leaning into video games during the COVID-19 pandemic to entertain and connect children with rare and chronic conditions, including cystic fibrosis. Click here to learn more.

What do you think of this news?

]]> ffc4052923cbd9e54fd63601f44302e0 MD forums Moderator started the discussion CAP-1002 Improves Muscle, Lung, Heart Function in Young DMD Patients, HOPE-2 Trial Data Show in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/cap-1002-improves-muscle-lung-heart-function-in-young-dmd-patients-hope-2-trial-data-show/ Tue, 26 May 2020 13:32:33 -0500 CAP-1002 Improves Muscle, Lung, Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

Treatment with CAP-1002 improves respiratory, cardiac and muscle function in boys and young men with advanced DMD, final data from the HOPE-2 trial show. Read more here.

What do you think of these findings?

]]> 535255f3314fb1254245e7baa8f1c935 MD forums Moderator started the discussion Unity and EU-wide Efforts Focus of Online Rare Disease Meet in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/unity-and-eu-wide-efforts-focus-of-online-rare-disease-meet/ Thu, 21 May 2020 15:45:07 -0500 Unity and EU-wide Efforts Focus of Online Rare Disease Meet

Some 1,500 delegates from 57 countries joined the virtual European Conference on Rare Diseases this month with a focus on unity and standardized policies. Read more about the conference here.

What are other important issues you think the global rare disease community should address?

]]> e0ba75992247894b443824c4e919f9f6 MD forums Moderator started the discussion Experts' Advice for Duchenne, Becker Patients Amid COVID-19 Pandemic in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/experts-advice-for-duchenne-becker-patients-amid-covid-19-pandemic/ Tue, 19 May 2020 13:46:57 -0500 Experts' Advice for Duchenne, Becker Patients Amid COVID-19 Pandemic

Neuromuscular specialists in the U.S. provided recommendations to help Duchenne and Becker MD patients deal with the challenges of the COVID-19 pandemic. Click here to read more.

Did you find these recommendations useful?

]]> e12300c04b8deab7e2aa7ac75aef19ff MD forums Moderator started the discussion Rare Diseases Research Network Opens Online Survey on COVID-19 in the forum COVID-19 and MD https://musculardystrophynews.com/forums/forums/topic/rare-diseases-research-network-opens-online-survey-on-covid-19/ Thu, 14 May 2020 17:40:23 -0500 Rare Diseases Research Network Opens Online Survey on COVID-19

The NIH-led research network hopes for 5,000 responses to its online survey of how people with rare diseases in the US are being affected by the pandemic. Click here to learn more about the survey and how you can participate in it.

How has the global pandemic affected you? What do you think the survey will reveal about the… Read more

]]> 584c8b3859525e7fc49e4e2ff89c4330 MD forums Moderator started the discussion NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/nord-webinar-outlines-covid-19-response-on-financial-policy-fronts/ Tue, 12 May 2020 21:41:37 -0500 NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Click here to read NORD’s call to action for rare disease patients on financial and legislative issues in the wake of the COVID-19 pandemic.

Which of these issues is most important to you and why?

]]> 9cf23d13756283f86bc03c4af29f6df3 MD forums Moderator started the discussion CureDuchenne Ventures Gives $1M to Support Non-viral Gene Therapy in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/cureduchenne-ventures-gives-1m-to-support-non-viral-gene-therapy/ Thu, 07 May 2020 16:22:29 -0500 CureDuchenne Ventures Gives $1M to Support Non-viral Gene Therapy

Click here to read about $1 million in seed funding to support work by Myosana Therapeutics on a potential non-viral and full-length dystrophin gene therapy for DMD patients.

What do you think of this news?

]]> 0038f8a5e75de2c9f98224eab2e81ce8 MD forums Moderator started the discussion In DMD, Increlex Improves Growth, but Not Muscle Function, Study Finds in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/in-dmd-increlex-improves-growth-but-not-muscle-function-study-finds/ Tue, 05 May 2020 16:05:49 -0500 In DMD, Increlex Improves Growth, but Not Muscle Function, Study Finds

Treatment for six months with Increlex hormone therapy improved growth but not muscle function in boys with DMD, an early study found. Learn more here.

What do you think of this study and its findings?

]]> 70fa0f72501ef7714b7fa3ef80ccca25 MD forums Moderator started the discussion DMD Mice See Improvements From AdipoRon Treatment, Study Shows in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/dmd-mice-see-improvements-from-adiporon-treatment-study-shows/ Thu, 30 Apr 2020 15:55:49 -0500 DMD Mice See Improvements From AdipoRon Treatment, Study Shows

AdipoRon, a potential new stategy to treat people with DMD, improved motor function and eased inflammation in a mouse model of the disease, a study found. Read more here.

What do you think of this news?

]]> 50844ff04b521930f531f49c74f693cb MD forums Moderator started the discussion In DMD Boys, Vyondys 53 Boosts Dystrophin Production, Early Data Show in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/in-dmd-boys-vyondys-53-boosts-dystrophin-production-early-data-show/ Tue, 28 Apr 2020 16:54:06 -0500 In DMD Boys, Vyondys 53 Boosts Dystrophin Production, Early Data Show

Vyondys 53 (golodirsen) treatment over 48 weeks increased dystrophin protein levels in boys with Duchenne muscular dystrophy, early trial results show. Read more about these findings here.

What do you think of this news?

]]> a92ead693ad9d50a6448191b3cb70e90 MD forums Moderator started the discussion MDA Let's Play Uses Gaming to Raise Funds for Neuromuscular Disorders in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/mda-lets-play-uses-gaming-to-raise-funds-for-neuromuscular-disorders/ Thu, 23 Apr 2020 14:39:16 -0500 MDA Let's Play Uses Gaming to Raise Funds for Neuromuscular Disorders

The Muscular Dystrophy Association is launching MDA Let’s Play, a platform to raise funding and awareness about neuromuscular disorders through gaming. Read more about it here.

What do you think of this news?

]]> bdfb270e63989917b3487c7728ab9a6b MD forums Moderator started the discussion Clinical Trial into Natural History of LGMD2i Enrolling in US, Denmark in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/clinical-trial-into-natural-history-of-lgmd2i-enrolling-in-us-denmark/ Tue, 21 Apr 2020 15:19:22 -0500 Clinical Trial into Natural History of LGMD2i Enrolling in US, Denmark

 

A study in 80 LGMD2i patients will use twice yearly clinical exams to better understand disease course, help with treatments, and validate a biomarker. Read more about the research here.

What do you think of this news?

]]> f8ffbbd322c13b11c959889c38b2ee8d MD forums Moderator started the discussion Pediatric Rare Disease Therapies on Rise, But Most Repurposed, Study Finds in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/pediatric-rare-disease-therapies-on-rise-but-most-repurposed-study-finds/ Thu, 16 Apr 2020 16:41:47 -0500 Pediatric Rare Disease Therapies on Rise, But Most Repurposed, Study Finds

The number of pediatric rare disease therapies has grown over the past decade, but most are repurposed old treatments, a study found. Read the full story here.

What do you think of this news?

]]> f874386c4fead8878b5f486f9df003e0 MD forums Moderator started the discussion DMD Patients in U.S. to Get Viltolarsen Under Expanded Access Program in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/dmd-patients-in-u-s-to-get-viltolarsen-under-expanded-access-program/ Tue, 14 Apr 2020 16:32:01 -0500 DMD Patients in U.S. to Get Viltolarsen Under Expanded Access Program

NS Pharma launched an expanded access program in the U.S. to allow certain Duchenne patients to gain access to viltolarsen. Click here to learn more about it.

What do you think of this plan?

]]> 43e7054d18c4b446e23fe855937a49ee MD forums Moderator started the discussion Machine Learning Helps Diagnose Specific MD Forms, Study Reports in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/machine-learning-helps-diagnose-specific-md-forms-study-reports/ Thu, 09 Apr 2020 13:37:31 -0500 Machine Learning Helps Diagnose Specific MD Forms, Study Reports

A tool using machine learning to identify specific MD subtypes form MRI scans was found to be accurate most of the time, according to a study. Read more about it here.

What do you think of this news?

]]> 67e9182e50409332c6482eb66c986d30 MD forums Moderator started the discussion Make the Most Out of Your Time - Lockdown Tips in the forum COVID-19 and MD https://musculardystrophynews.com/forums/forums/topic/make-the-most-out-of-your-time-lockdown-tips/ Tue, 07 Apr 2020 14:31:24 -0500 Make the Most Out of Your Time - Lockdown Tips

Click here to read Kevin Schaefer’s advice for making the most out of your time while self-isolating at home.

How are you spending your time at home?

]]> b5eeb2770bdf19a7e6b3d62f80c0a3d0 MD forums Moderator started the discussion Global Rare Disease Group's Focus: 1,000 New Therapies by 2027 in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/global-rare-disease-groups-focus-1000-new-therapies-by-2027/ Thu, 02 Apr 2020 16:32:11 -0500 Global Rare Disease Group's Focus: 1,000 New Therapies by 2027

Despite constraints due to the COVID-19 pandemic, the Paris-based IRDiRC continues efforts for 1,000 new therapies and better rare disease care by 2027. Read more here.

Do you think this goal is attainable? Why or why not?

]]> 3234f12f04d2b820eb987a1cd554cd98 MD forums Moderator started the discussion DMD Emflaza Supply Not Affected by COVID-19, PTC Therapeutics Says in the forum COVID-19 and MD https://musculardystrophynews.com/forums/forums/topic/dmd-emflaza-supply-not-affected-by-covid-19-ptc-therapeutics-says/ Tue, 31 Mar 2020 13:26:50 -0500 DMD Emflaza Supply Not Affected by COVID-19, PTC Therapeutics Says

Supplies of Emflaza, used for treating Duchenne muscular dystrophy, will not be affected by the COVID-19 pandemic, PTC Therapeutics said. Read more here.

Are you worried about how the COVID-19 pandemic might affect medical supplies and treatments for people with MD?

]]> d5c13a99e30dec6299b64ee80c531233 MD forums Moderator started the discussion Exosome Technology for DMD is Focus of Capricor Expansion in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/exosome-technology-for-dmd-is-focus-of-capricor-expansion/ Fri, 27 Mar 2020 16:21:52 -0500 Exosome Technology for DMD is Focus of Capricor Expansion

Capricor Therapeutics is expanding its program for exosome-based technologies currently being developed for the treatment of DMD. Click here to learn more about it.

Are you excited about this news?

]]> a3b089013d39174a78b7cc11f262a18c MD forums Moderator started the discussion Request to Approve Duchenne MD Exon 53 Skipping Treatment, Viltolarsen, Before FDA in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/request-to-approve-duchenne-md-exon-53-skipping-treatment-viltolarsen-before-fda/ Wed, 16 Oct 2019 17:47:32 -0500 Request to Approve Duchenne MD Exon 53 Skipping Treatment, Viltolarsen, Before FDA

An application before the FDA seeks approval of viltolarsen, a weekly IV infusion treatment for Duchenne muscular dystrophy amenable to exon 53 skipping. Read more about it here.

Are you excited about this news?

]]> 7c3b9a22366f934dec330d011372d9a5 MD forums Moderator started the discussion Fulfilling Work Doesn't Feel Like Work in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/fulfilling-work-doesnt-feel-like-work/ Tue, 15 Oct 2019 15:22:35 -0500 Fulfilling Work Doesn't Feel Like Work

Click here to read Hawken Miller’s column about how a fulfilling work experience has helped him find his purpose and improve his quality of life.

What do you do for work? Is it fulfilling?

]]> c3643ee1278f90bf50263a69ecdf4878 MD forums Moderator started the discussion Gene Therapy SRP-9003 Showing 'Very Encouraging' Results at 9 Months in Limb Girdle MD, Sarepta Reports in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/gene-therapy-srp-9003-showing-very-encouraging-results-at-9-months-in-limb-girdle-md-sarepta-reports/ Mon, 14 Oct 2019 17:10:20 -0500 Gene Therapy SRP-9003 Showing 'Very Encouraging' Results at 9 Months in Limb Girdle MD, Sarepta Reports

A 9-month update on three LGMD type 2E childen given one low-dose of the gene therapy SRP-9003 in a clinical trial shows “significant” gains in NSAD scores. Read more here.

What do you think of this study and its findings?

]]> ee844ca97c80ecbcb3c16ce5acd608db MD forums Moderator started the discussion Final Tests Planned into Potential OPMD Gene Therapy, Could Support Trial in Patients in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/final-tests-planned-into-potential-opmd-gene-therapy-could-support-trial-in-patients/ Fri, 11 Oct 2019 16:03:14 -0500 Final Tests Planned into Potential OPMD Gene Therapy, Could Support Trial in Patients

Benitec Biopharma’s plans 3 tests in a oculopharyngeal muscular dystrophy dog model, final steps to bringing its gene therapy, BB-301, into a Phase 1 trial. Click here to read more about this trial.

What do you think of this study and its findings?

]]> d8962c0b4fc9325dadf6b694824bad1a MD forums Moderator started the discussion NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/nord-2019-rare-disease-summit-set-for-oct-21-22-in-washington-dc/ Thu, 10 Oct 2019 15:02:18 -0500 NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

The 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit is set for Oct. 21-22 in Washington, D.C., with the theme “The Time is Now.” Click here to read more about it.

Did you know about this summit? Would you like to attend?

]]> 23d73ad144fe7e27c056603113ad4fc4 MD forums Moderator started the discussion PPMD Announces First Adult Certified Duchenne Care Center in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/ppmd-announces-first-adult-certified-duchenne-care-center/ Wed, 09 Oct 2019 16:11:32 -0500 PPMD Announces First Adult Certified Duchenne Care Center

Parent Project Muscular Dystrophy’s first Adult Certified Duchenne Care Center has the resources to address older patients’ needs. Read more about the Center here.

Have you found any of these resources helpful for you and your family?

]]> 43d68fa28457cd27ad28eeb89ec18582 MD forums Moderator started the discussion CureDuchenne Plans Oct. 11-13 'Futures' Meet at California's Disneyland in the forum Advocacy Work https://musculardystrophynews.com/forums/forums/topic/cureduchenne-plans-oct-11-13-futures-meet-at-californias-disneyland/ Tue, 08 Oct 2019 14:59:27 -0500 CureDuchenne Plans Oct. 11-13 'Futures' Meet at California's Disneyland

The CureDuchenne 2019 “Futures” conference, taking place Oct. 11-13 at the Disneyland Hotel in California, will focus on living and thriving with DMD. Click here to read more about it.

Did you know about this conference? Would you like to attend?

]]> f6ddfb7417eea169c018d5c35d832ef6 MD forums Moderator started the discussion Honoring Serena Lawrence: How Her Life Changed Mine in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/honoring-serena-lawrence-how-her-life-changed-mine/ Mon, 07 Oct 2019 15:00:02 -0500 Honoring Serena Lawrence: How Her Life Changed Mine

Leah Leilani reflects on the influence of a rare person who gave her the opportunity to speak out about living with mitochondrial myopathy. Click here to read more from Leah.

Do you have someone like this in your life?

]]> 6ace7d9f5e7ef032a96e710e18a00d1d MD forums Moderator started the discussion FDA Awards $1M to Duchenne MD Phase 2 Study of Ifetroban in Treating Related Heart Disease in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/fda-awards-1m-to-duchenne-md-phase-2-study-of-ifetroban-in-treating-related-heart-disease/ Fri, 04 Oct 2019 16:58:38 -0500 FDA Awards $1M to Duchenne MD Phase 2 Study of Ifetroban in Treating Related Heart Disease

The $1 million grant to Cumberland Pharmaceuticals is going to support a soon-to-open 12-month trial of oral ifetroban in DMD patients with heart muscle damage. Click here to read the whole story.

Are you excited about this news?

]]> 3a961315661861a43bd50a2eba4291c9 MD forums Moderator started the discussion Final Tests Planned into Potential OCMD Gene Therapy, Could Support Trial in Patients in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/final-tests-planned-into-potential-ocmd-gene-therapy-could-support-trial-in-patients/ Thu, 03 Oct 2019 15:22:28 -0500 Final Tests Planned into Potential OCMD Gene Therapy, Could Support Trial in Patients

Benitec Biopharma’s plans 3 tests in a oculopharyngeal muscular dystrophy dog model, final steps to bringing its gene therapy, BB-301, into a Phase 1 trial. Read more here.

What do you think about this research project?

]]> a07c9bde7a21f5faa5616e7a3cb2a54d MD forums Moderator started the discussion Redefining the Word 'Independence' in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/redefining-the-word-independence/ Wed, 02 Oct 2019 14:38:15 -0500 Redefining the Word 'Independence'

For Hawken Miller, ‘independence’ is more about mental autonomy rather than being physically self-sufficient. Click here to read more from Hawken.

What does independence look like for you? Do you want more independence?

]]> be70376c4f980b13c60387f677e8f80b MD forums Moderator started the discussion Here's Your One-Stop Informative Guide on Durable Medical Equipment in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/heres-your-one-stop-informative-guide-on-durable-medical-equipment/ Tue, 01 Oct 2019 14:22:53 -0500 Here's Your One-Stop Informative Guide on Durable Medical Equipment

Click here to access CureDuchenne’s comprehensive, interactive guide to help families easily find and choose well among equipment needed for every stage of Duchenne MD.

Did you find this guide to be a helpful resource for you and your family?

]]> 74a6e718cb4676c2546c8b97f35b0728 MD forums Moderator started the discussion FDA Fast Tracks Suvodirsen Designation in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/fda-fast-tracks-suvodirsen-designation/ Mon, 30 Sep 2019 13:22:11 -0500 FDA Fast Tracks Suvodirsen Designation

The U.S. Food and Drug Administration has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). Read more about the decision here.

Are you excited about this news?

]]> 362562d9d5b29aac8143b745c8ba74ff MD forums Moderator started the discussion National LGMD Conference Builds the MD Community in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/national-lgmd-conference-builds-the-md-community/ Fri, 27 Sep 2019 15:16:06 -0500 National LGMD Conference Builds the MD Community

Click here to read Ralph Yaniz’s column about the highlights of a recent LGMD conference, where participants had the opportunity to build community.

Do you live with LGMD? If so, do you feel engaged with your community?

]]> 82f9ed82bbd835a78d61d7ea9a657386 MD forums Moderator started the discussion Review Highlights Microdystrophin Gene Therapy in IGNITE DMD trial in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/review-highlights-microdystrophin-gene-therapy-in-ignite-dmd-trial/ Thu, 26 Sep 2019 14:15:14 -0500 Review Highlights Microdystrophin Gene Therapy in IGNITE DMD trial

A review study highlights the benefits of adding an extra protein domain – neuronal nitric oxide synthase (nNOS) – to microdystrophin gene therapy. Click here to read more about it.

What do you think about this study and its findings?

]]> d5131bce0c51d5ab11cbb5362b37346b MD forums Moderator started the discussion Researchers Identify New Blood DMD Biomarkers in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/researchers-identify-new-blood-dmd-biomarkers/ Wed, 25 Sep 2019 14:12:38 -0500 Researchers Identify New Blood DMD Biomarkers

Researchers have identified a new set of blood biomarkers of Duchenne muscular dystrophy and of response to standard glucocorticoid therapy. Find out more about this research discovery here.

What do you think of this news?

]]> a31e0d551d2dad5f36d7a56cc2f1a49a MD forums Moderator started the discussion Acceleron Stops Clinical Development of ACE-083 for FSHD in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/acceleron-stops-clinical-development-of-ace-083-for-fshd/ Tue, 24 Sep 2019 16:21:45 -0500 Acceleron Stops Clinical Development of ACE-083 for FSHD

Acceleron Pharma will stop clinical development of its ACE-083 candidate for facioscapulohumeral muscular dystrophy due to lack of efficacy, officials said. Read more about it here.

What do you think of this news?

]]> f04fc1fd6dbc8e8d220258bd9aade5a1 MD forums Moderator started the discussion 30 Days of MD' Is Patient-centered Awareness Lasting All Year in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/30-days-of-md-is-patient-centered-awareness-lasting-all-year/ Mon, 23 Sep 2019 14:20:54 -0500 30 Days of MD' Is Patient-centered Awareness Lasting All Year

Click here to read more about 30 Days of MD, a series of stories written by patients, caregivers, therapists, researchers, and others to raise awareness about muscular dystrophy.

Have you been following 30 Days of MD? What are some of your favorite stories from the initiative?

]]> cfbb2701a2eaee30814b89cc54e54e02 MD forums Moderator started the discussion $6.6M Awarded to DMD and Similar Research Projects in the forum Diagnosis​ ​Info​ ​and​ ​General​ ​Questions https://musculardystrophynews.com/forums/forums/topic/6-6m-awarded-to-dmd-and-similar-research-projects/ Fri, 20 Sep 2019 16:00:25 -0500 $6.6M Awarded to DMD and Similar Research Projects

The Muscular Dystrophy Association has awarded $6.6 million support research into causes of muscular dystrophies and like disorders, and advance treatments like gene therapy. Read the full story here.

What do you think of this news? Which of these research projects are you most excited about?

]]> c59fb54a8b6224691fd9706be3941b59 MD forums Moderator started the discussion My Disability Does Not Mean I Am Broken in the forum Adults​ ​With​ ​Muscular Dystrophy https://musculardystrophynews.com/forums/forums/topic/my-disability-does-not-mean-i-am-broken/ Thu, 19 Sep 2019 15:59:18 -0500 My Disability Does Not Mean I Am Broken

Leah Leilani argues that, despite her disability, she is not broken. If anything, her disability adds to her value as a human being. Click here to read more from Leah.

Do you agree with Leah? Has anyone ever described you as “broken”? How did you respond?

]]>