Formed in September 2013, the LGMD2D Foundation is a registered 501(c)3 non-profit foundation built for families living with LGMD2D - both patients and caregivers - by families with the same diagnosis. We, too, saw there was a gap in providing information and resources to those living with this rare disorder. Our mission is to expedite the development of treatments or a cure for Limb-Girdle Muscular Dystrophy, type 2D / R3 (LGMD2D).
LGMD2D Foundation的外部链接
20 SUNNYSIDE AVE
us，California，Mill Valley，94941
Looks incredible, Sarepta Therapeutics!! Thank you for your continued partnership and all you do for the LGMD community. ??
We’re lighting up green for limb-girdle muscular dystrophy! Today, our Genetic Therapies Center of Excellence in Columbus, Ohio is glowing with green lights for LGMD Awareness Day. We’re shining bright to show our support for the LGMD community. #LGMD2024
???? Today is LGMD Awareness Day!! ???? Today we come together, on the 10th Anniversary of LGMD Awareness Day, to love, honor and raise awareness for all patients around the globe living with Limb-Girdle Muscular Dystrophy (LGMD). We are thankful for the LGMD Awareness Foundation, Inc. for making this day recognized around the world. We are so proud of the work this amazing community has done over the past 10 years and look forward to what's ahead for us in the future. There is so much promise for this entire community and the much needed treatments these patients deserve. The LGMD2D Foundation, today and everyday, will continue to raise awareness, advocate and push for treatments for our LGMD2D/R3 patients. Learn more about LGMD2D: https://lgmd2d.org/
Great opportunity to hear from great minds on gene therapy this upcoming learning series from the Muscular Dystrophy Association. These sessions are free and are bound to be informative for the future of rare disease treatments.
Join us on August 29, September 5, and September 12 for this in-depth learning series all about advancements and considerations in gene therapy across neuromuscular diseases. This multi-day series is free for people living with neuromuscular disease and their caregivers. Presentations from experts in the field include: Craig Zaidman, MD and Natalie Goedecker CPNP,?Washington University School of Medicine in St. Louis, Barry Byrne, MD, PhD?UF Health, Michael Storey, PharmD from?Nationwide Children's Hospital, Paul Melmeyer, VP of Public Policy and Advocacy at Muscular Dystrophy Association, and Aravindhan Veerapandiyan, MD from?Arkansas Children's. Each presentation will also offer a live Q&A. REGISTER HERE: ?https://lnkd.in/gv-AG34k #MDA #GeneTherapy #MuscularDystrophy #Neuromuscular #Duchenne #SpinalMuscularAtrophy #SMA
Congratulations Kat Bryant Knudson on your recent nomination for the RARE Champions of Advocacy award nomination! You have done so much for the entire LGMD community. Our partnership & driving change for patients together is invaluable. Way to go!! ????
Congratulations to Kat Bryant Knudson, founder and CEO of The Speak Foundation and editor-in-chief of LGMD News magazine! ?? Kathryn has been nominated for Global Genes "RARE Champion in Advocacy" Award. This award recognizes an individual rare disease patient or advocate who has created innovative programming and/or successful partnerships to support, educate, research, or enable their communities in meaningful and impactful ways. Without a doubt, Kathryn would be well deserving of this award! Her dedication and commitment to the LGMD community is outstanding. ? https://lnkd.in/gHnQeecy #CureLGMD #TogetherWeAreStronger #LgmdAwareness #globalgenes
Land of the free because of the brave. Remembering their courage, honor and bravery, today and every day. #memorialday #unitedstates #americathebeautiful #honoringourheroes
So wonderful to see such an amazing and dedicated person receive such a wonderful honor. Dr. Mendell is very dear to the LGMD community, especially for LGMD2D, and our community is forever grateful for his devotion to muscular dystrophy. He is critical to where we are today with gene therapy R&D. This honor is beyond deserved. ????
Congratulations to Jerry Mendell, MD, retired neurologist and principal investigator, and current senior advisor in the Center for Gene Therapy at Nationwide Children's, for being named one of TIME's 100 Most Influential People in global health. Two FDA-approved gene therapies have come from Dr. Mendell's lab: Zolgensma for spinal muscular atrophy in 2019 and Elevidys for Duchenne muscular dystrophy in 2023. Dr. Mendell is a fellow in the National Academy of Medicine in 2021. He was also the first recipient of the American Society for Gene and Cell Therapy (ASGCT)’s Jerry Mendell Translational Medicine Award. He has published more than 400 articles and authored books on skeletal muscle disease, peripheral nerve disorders, and gene therapy. Learn more: https://bit.ly/49ZD7DR #TIME100Health
Meet Donavon. A very special member of our LGMD2D/R3 community. Donavon has been a pioneer for treatment progress in muscular dystrophy and we are so honored to be able to share his story as our latest People of LGMD2D post. ?? Thank you, Donavon, for all you have done for LGMD research, advocacy and awareness. #lgmd2d #lgmd #lgmdawareness #curelgmd #StrongerTogether
The 10th annual LGMD AWARENESS DAY will be celebrated on September 30, 2024. ?? This year the theme is "A Decade of Difference". We are excited to be creating an awareness toolkit to help the LGMD community Advocate - Educate - Celebrate limb-girdle muscular dystrophy. ? Be the First to Know When the Toolkit Drops! Sign up for the toolkit at https://lnkd.in/gwUz8BAE #TogetherWeAreStronger #LGMDawareness
we can't wait, LGMD Awareness Foundation, Inc. !! ????
The 10th annual LGMD AWARENESS DAY will be celebrated in just 6 months on September 30, 2024. This year the theme is "A Decade of Difference". We are excited to be creating an awareness toolkit to help the LGMD community Advocate - Educate - Celebrate limb-girdle muscular dystrophy. Be the First to Know When the Toolkit Drops! Sign up for the toolkit at https://lnkd.in/gwUz8BAE #TogetherWeAreStronger #LGMDawareness
This is exciting news for the LGMD community and the sarcoglycanopathies. Keep pushing LGMD treatments, Atamyo Therapeutics. And thank you for your focus on LGMD!
Atamyo Therapeutics?announces it has obtained regulatory authorization in Europe to initiate a clinical trial for ATA-200, its gene therapy to treat limb-girdle muscular dystrophy type 2C/R5.?This authorization was first granted by the Italian Medicines Agency AIFA Agenzia Italiana del farmaco, then by the French Medicines Agency ANSM Agence nationale de sécurité du médicament et des produits de santé. Highlights: --Atamyo has received approval to initiate a clinical trial of ATA-200 gene therapy in France and Italy --ATA-200 is a single-injection gene therapy aimed to treat LGMD2C/R5 caused by mutations in the g-sarcoglycan gene --The phase 1b, dose-escalation study will evaluate the safety and efficacy of ATA-200 in children “We are thrilled to obtain our CTA approval in France and Italy for the devastating LGMD2C/R5 disease affecting primarily a pediatric population and for which there is no approved treatment,” said Dr?Sophie Olivier,?Chief Medical Officer of Atamyo. “Atamyo plans to initiate dosing in patients for ATA-200 in third quarter 2024”. “LGMD 2C/R5 is a severe muscular dystrophy with an onset in early childhood and loss of ambulation generally occurring before adolescence,” said Pr. Giacomo Comi, Full Professor of Neurology at the University of Milan (Italy), and principal investigator of this trial. “It is a great motivation to know that the work we are doing has the potential to make a life-changing difference for the patients affected by this disease.” “This is a significant milestone for LGMD-2C/R5 patients and for Atamyo as ATA-200 is the first treatment targeting LGMD-2C/R5 to enter clinical trials,” said?Stephane Degove, CEO of Atamyo Therapeutics. “With the ongoing clinical trial of ATA-100 in LGMD2I/R9, the initiation of the clinical program for ATA-200 confirms our unique capabilities in bringing to patients suffering from?limb-girdle muscular dystrophies?a new generation of safe and effective gene therapies.” ATA-200, a gene therapy candidate for LGMD2C/R5, delivers a normal copy of the gene for production of g-sarcoglycan protein. In preclinical models, a single systemic injection of ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology. ATA-200 has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA) The therapy is based on the research of Atamyo Chief Scientific Officer?Isabelle Richard, Ph.D., Research Director at National Center of Scientific Research (CNRS) in France and head of the Progressive Muscular Dystrophies Laboratory at?Genethon. #clinicaltrials #genetherapy #lgmd To read the complete press release:? https://lnkd.in/gMbUXK9c