MDF Magazine Issue 72 December 2023

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Thank you <strong>MDF</strong>SA Mukovhe Maleri I would like to thank all the people who helped me to get the wheelchair you gave me. It's really helpful to me because I can go anywhere I want without anyone having to accompany me. I really appreciate it as I will be able to further my education. I hadn't been able to go to school, but now I don't have any problem with that because of the wheelchair. I am very appreciative. Bongile Booi Thank you to the Muscular Dystrophy Foundation for your help with the wheelchair. I am now able to move around on my own. God bless you. Noxolo Thabethe My name is Noxolo Thabethe, and I would like to say thank you very much to <strong>MDF</strong>SA and Wheelchairs on the Run for the new electric wheelchair that I received last week. Gugulethu Shongwe I just want to thank you so much for your help – the past five years were the most difficult time for me without having a wheelchair. It was hard for me even to move in the yard and I was always indoors. Thanks to you I now can move and go anywhere I want to go. Please don't stop giving love and help as we really appreciate it. Thank you from the bottom of my heart. The Hidden Pain By Mandy Martin In the depths of a parent's heart, a pain so profound, To witness your children, with MD, bound. With every step I’ll lend a hand, To help you rise, and help you stand I’ll hold you close when tears are shed, And will give my all to help you take the next step Helpless I have to witness your fight, All that I can do is hold you tight I have to watch your strength decline, I yearn for a miracle to make it mine A mix of emotions, a storm within, How can I describe my feelings of sin But this demon is relentless, its grip hard to restrain. Your strength is unmatched, a beacon of light, But my hear breaks silently, hidden from sight I sit in a corner, shedding tears in silence, In solitude I cower, weeping with quiet defiance. In the face of struggles, a parent's heart does ache, To witness their child's pain, their every step they take. Your spirit, your fight dissolving, fading from sight, Cuts through me like a jiggered knife I wish I could bear all your pain And trade places with you to take your strain The burdens that weighing you down, Together, we'll conquer and turn it around. I long to protect you, to shield you from pain, The Muscular Dystrophy Foundation of SA would like to thank Sarepta Therapeutics for the grant towards Awareness and genetic testing for people diagnosed with LGMD.
California’s stem cell agency funds the potential gene editing DMD therapy (SACRAMENTO) UC Davis Professor Aijun Wang and his team are collaborating with Murthy laboratory at UC Berkeley to develop a much-needed cure for Duchenne muscular dystrophy (DMD). They are designing a therapy to treat DMD before birth by editing the gene that encodes dystrophin, a key protein in stabilizing muscle fiber. “We are developing a gene editing therapy that would allow pregnant mothers to give birth to children who are free from DMD,” said Wang, professor of surgery and biomedical engineering. Wang is the vice chair for translational research, innovation and entrepreneurship at the Department of Surgery and co-directs the Center for Surgical Bioengineering at UC Davis. He also leads the Wang Lab, a prime research hub in stem cell therapy and gene editing for early treatments of birth defects such as spina bifida. This groundbreaking work is funded by a $2 million Quest Award from the California Institute for Regenerative Medicine (CIRM). The DISC-2 Quest Awards Program promotes the discovery of promising new stem cell-based and gene therapy technologies that could lead to broad use and improved patient care. The muscle weakness in DMD patients becomes increasingly noticeable between the ages of 3 and 5. By the age of 12, most patients require a wheelchair. By adolescence, the patient’s heart and breathing muscles weaken, leading to serious, life-threatening complications. About Cure Duchenne Muscular Dystrophy (Cure DMD) The team is developing a new technology called “Cure Duchenne Muscular Dystrophy (Cure DMD).” The hope is to treat DMD before birth by editing the genes that encode, or cause the production of, dystrophin. The plan is to edit these genes in the heart, diaphragm and limb muscles in utero and correct the DMD mutations before the onset of the disease. Overview of the study design. LNPs=lipid nanoparticles Most DMD patients would need a wheelchair What is Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy is a rare genetic disorder causing muscle loss and physical impairments in young people. It is characterized by muscle fiber degeneration due to a gene mutation that prevents the body from producing fully functional dystrophin. Dystrophin protein plays a critical role in stabilizing the muscle membrane when muscles contract. Without it, muscle cells become damaged and weak. Cure DMD will be delivered via an in-utero injection to the fetus diagnosed with DMD. It will use a non-viral delivery method known as lipid nanoparticle to transfect, or introduce, cells using mRNA. This transfection causes the temporary expression of an enzyme that does the gene editing. The Wang and Murthy labs recently showed the feasibility of this approach in a study published in Bioactive Materials. “Gene editing provides a permanent cure as it is a correction of the mutation,” Wang explained. “When the treatment is offered before birth, and if it is done right with mutation efficiently edited, one can prevent the disease from happening.” The team will first test Cure DMD by giving an in-utero injection to a pregnant DMD mouse model. They will evaluate the safety of the treatment to the developing fetuses and pregnant female mice and monitor the edited mice for any birth defects.
Page 1 and 2: Summer Issue 72 December 2023 MAGAZ
Page 3 and 4: MDF support information To learn mo
Page 5 and 6: Why bother getting a FREE genetic t
Page 7 and 8: Meet the Gauteng branch chairperson
Page 9 and 10: Celebrating World Duchenne Awarenes
Page 11 and 12: KZN branch reaches out to the commu
Page 13 and 14: and gifts. • Solly Manjra Caterer
Page 15: Marking Casual Day 2023 By Madelein
Page 19 and 20: 5 things we’ve learned about find
Page 21 and 22: By Editorial Team, Spinal Muscular
Page 23 and 24: Corinne Grgas By Rebecca Hume Muscu
Page 25 and 26: adjust her schedule when she felt c
Page 27 and 28: park. Later in the trip, on the Auo
Page 29 and 30: day again. I certainly have never s
Page 31 and 32: family member, or coworker whom you
Page 33 and 34: Random gravity check... By Andrew M

MDF Magazine Issue 72 December 2023

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