肌肉萎縮症協會授予近 500 萬美元的新撥款，用於資助肌肉萎縮症及相關肌肉疾病的研究

肌肉營養不良協會 (MDA) 已授予近兩打新撥款，用於資助各種形式的肌肉營養不良的研究，包括杜氏肌肉營養不良症和肢帶肌肉營養不良症，以及相關的肌肉疾病。

The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases.

肌肉營養不良協會 (MDA) 已授予近兩打新撥款，用於資助各種形式的肌肉營養不良的研究，包括杜氏肌肉營養不良症和肢帶肌肉營養不良症，以及相關的肌肉疾病。

Altogether, the funding totals more than $5 million across 21 grants, according to a press release from the nonprofit.

根據該非營利組織的新聞稿，21 項贈款的資金總額超過 500 萬美元。

“The Muscular Dystrophy Association is proud to continue driving critical research that brings hope and tangible progress to the neuromuscular community,” said Donald S. Wood, PhD, president and CEO of the MDA. “These projects reflect our dedication to the people we serve; we are eager to see how they will fuel clinical and scientific progress and, ultimately, lead to new treatments.”

「肌肉營養不良協會很自豪能夠繼續推動關鍵研究，為神經肌肉界帶來希望和切實進展，」MDA 總裁兼首席執行官唐納德·S·伍德博士說。 「這些項目體現了我們對我們所服務的人們的奉獻精神；我們渴望看到它們將如何推動臨床和科學進步，並最終帶來新的治療方法。

There are 14 research grants in all, as well as seven development awards for fellowship trainees. The MDA partnered with two other nonprofits on three collaboration grants — one for a University of California Los Angeles researcher seeking to develop a gene therapy for a type of limb-girdle muscular dystrophy (LGMD).

共有 14 項研究資助，以及 7 項針對實習生的發展獎。 MDA 與另外兩個非營利組織合作提供了三項合作資助，其中一項資助給加州大學洛杉磯分校的一名研究人員，旨在開發一種針對肢帶型肌肉營養不良症(LGMD) 的基因療法。

Over $5M in funding awarded across 21 MDA grants

21 項 MDA 撥款超過 500 萬美元

The newly awarded funding includes several research grants to well-established scientists. For example, Jyoti Jaiswal, PhD, a professor at Children’s National Research and Innovation, a hospital in Washington, D.C., won funding for his work to prevent muscle cell death and damage in Duchenne muscular dystrophy, known as DMD.

新授予的資金包括向知名科學家提供的幾項研究資助。例如，華盛頓特區兒童國家研究與創新醫院的教授 Jyoti Jaiswal 博士因其預防杜氏肌肉營養不良症（DMD）肌肉細胞死亡和損傷的工作而獲得了資助。

DMD is caused by gene mutations that lead to a lack of dystrophin — a protein that normally acts like a shock absorber in muscle cells to limit damage during muscle contractions. Without dystrophin, muscles accumulate more wear and tear damage over time, ultimately driving disease symptoms. Jaiswal’s project will explore a therapy that targets fat-like molecules to stabilize muscle cell membranes, with the goal of reducing muscle damage and improving function in DMD.

DMD 是由導致肌肉營養不良蛋白缺乏的基因突變引起的，這種蛋白通常在肌肉細胞中起到減震器的作用，以限制肌肉收縮期間的損傷。如果沒有肌肉營養不良蛋白，肌肉會隨著時間的推移累積更多的磨損和撕裂損傷，最終導致疾病症狀。 Jaiswal 的計畫將探索一種針對脂肪類分子來穩定肌肉細胞膜的療法，其目標是減少肌肉損傷並改善 DMD 的功能。

Another of the newly funded projects will explore the use of gene-editing technologies to treat LGMD, a group of genetic disorders characterized by muscle wasting in the shoulders and hips. That project, led by Scot Wolfe, PhD, of the University of Massachusetts Chan Medical School, will be testing gene editing to correct mutations that cause LGMD type 2G/R7. Wolfe is using stem cells, a mouse model, and other approaches to develop a platform that could also be expanded to treat other types of LGMD.

另一個新資助的計畫將探索使用基因編輯技術來治療 LGMD，這是一組以肩膀和臀部肌肉萎縮為特徵的遺傳性疾病。該計畫由馬薩諸塞大學陳醫學院的 Scot Wolfe 博士領導，將測試基因編輯以糾正導致 LGMD 2G/R7 型的突變。 Wolfe 正在使用幹細胞、小鼠模型和其他方法來開發一個平台，該平台也可以擴展到治療其他類型的 LGMD。

A separate project focused on LGMD is being jointly funded by the MDA and the Coalition to Cure Calpain 3. That project, led by Melissa Spencer, PhD, co-director of the Center for Duchenne Muscular Dystrophy at UCLA, aims to develop a gene therapy for LGMD type 2A/R1. This type of LGMD is caused by mutations in the CAPN3 gene; the project is focused on finding effective ways to deliver a healthy version of this gene to muscle cells.

MDA 和治癒 Calpain 3 聯盟共同資助了一個專注於 LGMD 的獨立計畫。型。這種類型的 LGMD 是由 CAPN3 基因突變引起的；該計畫的重點是尋找有效的方法將該基因的健康版本傳遞給肌肉細胞。

“We are excited to support Dr. Spencer’s groundbreaking work in advancing gene therapy for LGMD2A/R1,” Jennifer R. Levy, PhD, scientific director of the Coalition to Cure Calpain 3, said in a separate press release. “By funding this project, we are not only pushing the boundaries of what’s possible in genetic research but also reinforcing our commitment to finding life-altering treatments for those affected by this rare condition. This collaboration with MDA will enable us to accelerate progress and bring hope to the LGMD2A/R1 community.”

「我們很高興能夠支持 Spencer 博士在推進 LGMD2A/R1 基因治療方面的開創性工作，」治愈 Calpain 3 聯盟的科學主任 Jennifer R. Levy 博士在另一份新聞稿中表示。 「透過資助這個項目，我們不僅突破了基因研究的可能界限，而且加強了我們為受這種罕見疾病影響的人尋找改變生活的治療方法的承諾。與 MDA 的合作將使我們能夠加快進展，並為 LGMD2A/R1 社群帶來希望。

Spencer said the new funding “will allow us to refine our gene therapy approach and move closer to a treatment that could significantly improve the quality of life for people living with LGMD 2A/R1.”

Spencer 表示，新資金“將使我們能夠完善基因治療方法，並更接近能夠顯著改善 LGMD 2A/R1 患者生活品質的治療方法。”

According to Sharon Hesterlee, PhD, the MDA’s chief research officer, this work by Spencer “represents a major step forward in gene therapy” for this type of LGMD.

MDA 首席研究官 Sharon Hesterlee 博士表示，Spencer 的這項工作「代表了此類 LGMD 基因治療的重大進步」。

“Our partnership with C3 highlights the importance of collaboration in advancing research and finding potential cures for neuromuscular diseases,” Hesterlee said.

「我們與 C3 的合作凸顯了合作在推進研究和尋找神經肌肉疾病潛在治療方法方面的重要性，」Hesterlee 說。

Other projects funded by the MDA in this round will focus on neuromuscular conditions like spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

本輪 MDA 資助的其他計畫將重點放在脊髓性肌肉萎縮症 (SMA) 和肌萎縮側索硬化症 (ALS) 等神經肌肉疾病。

The MDA also has awarded more than a half-dozen grants to support early-career researchers studying muscular dystrophy and related disorders, with project goals ranging from making gene therapy safer to evaluating the effects of hormones in specific types of muscular dystrophy

MDA 也授予了超過六項撥款，以支持研究肌肉營養不良症和相關疾病的早期職業研究人員，計畫目標包括使基因治療更安全，到評估荷爾蒙對特定類型肌肉營養不良症的影響

“We are excited to award these new grants to researchers whose work has the potential to bring us closer to disease-modifying treatments for neuromuscular diseases,” Hesterlee said. “These grants not only support established researchers but also nurture the next generation of scientists who will continue the fight against these debilitating diseases.”

海斯特利說：“我們很高興將這些新的資助授予研究人員，他們的工作有可能使我們更接近神經肌肉疾病的疾病緩解治療。” 「這些贈款不僅支持現有的研究人員，還培養下一代科學家，他們將繼續與這些使人衰弱的疾病作鬥爭。”

The MDA’s investment to date in neuromuscular disease research exceeds $1 billion, according to the association.

據該協會稱，MDA 迄今為止在神經肌肉疾病研究方面的投資超過 10 億美元。