World Without GNE Myopathy

09/04/2025

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This is the story of Prasanna and his daughter Nidhi, a patient of Pompe Disease. Written by Prof. Sudha Bhattacharya, WWGM.

Read in full at gne-myopathy.org/post/applying-management-principles-to-rare-diseases-how-a-dad-navigated-daughter-through-pompe-disease

Long before the professional management degree became a passport to success, the world of commerce was managed (perhaps better) by intuitive managers. With a macro vision of the world around them, and a micro vision of their own immediate circumstances, an intuitive manger has the innate talent to d...

10/03/2025

WWGM says ! ❤️

08/03/2025

🎤 🌏 Meet the incredible global leaders shaping the future of at the WWGM Annual Conference March 8 & 9, ‘25! 🧬💡

Dr Paul Martin is at the forefront of developing cutting edge gene therapies for a number of rare neuromuscular diseases including GNE myopathy.

Ms Michela Onali is a winner of the EURORDIS Black Pearl award for her exceptional contributions to advocacy and research for GNE myopathy and other rare diseases.

Dr. Jin-Hong Shin is a neurologist with interest in neuromuscular disorders. He has worked and published extensively on GNE myopathy and has successfully conducted a clinical trial to test the efficacy of 6’-sialyllactose for treating GNE myopathy.

Dr. Wakako Yoshioka is a GNE myopathy patient and a clinical researcher. She has published 18 research papers on GNE myopathy and other neuromuscular disorders. Her work has helped us better understand the progression of GNE myopathy and its variability.

To know more about this event and register please visit gne-myopathy.org/annual-conference-25

07/03/2025

🎤 Meet the incredible speakers shaping the future at the WWGM Annual Conference March 8 & 9, ‘25! 🧬💡

Dr Arkasubhra Ghosh has developed a gene therapy platform to treat inherited eye diseases and is working on developing a similar therapy for rare muscle diseases.

Dr. Ravi Manjithaya is investigating the role of cellular degradative systems in the pathology of rare genetic disorders, and their potential use to find new drug targets.

Ms. Divya Rao is doing her Ph.D. under the supervision of Dr. Munia Ganguli who is one of the few scientists developing non-viral gene delivery systems for gene therapy to muscle cells for treating GNE myopathy and other rare diseases.

For mor information and to register for this event please visit www.gne-myopathy.org/annual-conference-25

06/03/2025

🎤 🩺 Meet the incredible speakers shaping the future at the WWGM Annual Conference March 8 & 9, ‘25! 🧬💡

Dr. Vishnu V Y runs a clinic on comprehensive neuromuscular disorders and was a fellow of the International Centre for Genomic Medicine in Neuromuscular Diseases where AIIMS was a participating institute.

Dr. Satish Khadilkar is one of the first Indian neurologists to develop extensive expertise in neuromuscular disorders. He was the first to identify a number of Limb Girdle Muscular Dystrophies including the ‘Agarwal’ mutation in calpainopathies and the Rajasthani founder mutation in GNE myopathy.

Dr. Soaham Desai is working to provide super speciality neurological services to the under-privileged, especially those coming from rural areas and to increase community awareness of neurological disorders.

For more information or to register visit https://www.gne-myopathy.org/annual-conference-25

05/03/2025

Less than a week to go!

🌿 Voices of Change: WWGM Annual Conference 2025 🎤✨

Meet the incredible speakers shaping the future at the WWGM Annual Conference ‘25! 🧬💡 Join us on March 8th & 9th as experts from around the world come together to share insights, research, and innovations. Over the next few days we will be introducing you to our incredible speakers! 🔬🌎

Dr. Rakesh Mishra is a distinguished scientist with extensive experience in genomics and gene regulation. He is spearheading the team at TIGS to develop mRNA therapies for rare genetic diseases.

Dr Nalini, NIMHANS, Bangalore is an extremely experienced neurologist who diagnosed the first patient with GNE myopathy in India and has one of the most extensive experience of seeing patients with GNE myopathy.

Dr Rajesh Iyer, Tata Institute for Genetics and Society, India is the foremost scientist in the team at TIGS developing mRNA therapeutic platforms for treatment of rare genetic disorders affecting muscles.

Dr. Seena Vengalil specializes in neuromuscular diseases and has tremendous research and clinical experience with more than 100 peer reviewed publications. She is presently conducting a natural history study of .

To register for this free online event visit https://www.gne-myopathy.org/annual-conference-25

02/03/2025

🌿 Voices of Change: WWGM Annual Conference 2025 🎤✨

Meet the incredible speakers shaping the future at the WWGM Annual Conference ‘25! 🧬💡 Join us on March 8th & 9th as experts from around the world come together to share insights, research, and innovations.

Stay tuned for speaker highlights! 🔬🌎

01/03/2025

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Only one week to go for WWGM's Annual Conference. It's online and free for anyone to attend. Expect to hear from various scientific, medical and other experts on and on cutting edge work and developments. Get up to date on all things GNE Myopathy. Sessions start at 3 PM IST on March 8.

To register please visit https://www.gne-myopathy.org/annual-conference-25

28/02/2025

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Today, we stand together in support of those affected by rare diseases. 💙💪 Rare Disease Day reminds us to spread awareness, encourage research, and show solidarity with the rare disease community. Every voice matters, and every story deserves to be heard. Let’s continue to believe in hope, healing, and a brighter future! ✨

🌿 Believe there is hope for healing
📅 28 February 💙

27/02/2025

Please support the mission of creating a World Without GNE Myopathy by donating to our and efforts for . Even a little bit goes a long way!

26/02/2025

Chapter 4 - Cure

When people living with rare diseases and their loved ones come together, they can forge a path towards a cure.

🖌️ by Pixel Trove

21/02/2025

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"Our does not only take a toll on our physical health but also on our ."

🎨 by