LGMD

Registration Now Open for 2024 MDA Clinical & Scientific Conference, to Showcase Cutting Edge Research Advancements and Clinical Achievements in Neuromuscular Disease

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Wednesday, August 9, 2023
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New York, Aug. 09, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA), convener of the largest worldwide gathering of the neuromuscular disease community, announced today that registration is now open for the 2024 MDA Clinical & Scientific Conference being held March 3-6, 2024, at the Hilton Orlando in Orlando, Florida.

Key Points: 
  • New York, Aug. 09, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA), convener of the largest worldwide gathering of the neuromuscular disease community, announced today that registration is now open for the 2024 MDA Clinical & Scientific Conference being held March 3-6, 2024, at the Hilton Orlando in Orlando, Florida.
  • The four-day international event will bring together renowned researchers, clinicians, academicians, advocates, and industry leaders to explore the latest advances in the field of neuromuscular disease.
  • We are encouraging more participation from allied health providers in addition to the MDA Care Center Network medical teams, and the research and advocacy communities,” said Donald S. Wood, Ph.D., President & CEO, MDA.
  • 2024 MDA Clinical & Scientific Conference Social Media Toolkit for registrants, panelists, supporters/sponsors, and advocacy organizations, is available here .

Promising disease modifying approach to Duchenne muscular dystrophy with Neu-REFIX® Beta 1,3-1,6 glucan* from Japan; the first such clinical report.

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Friday, August 4, 2023
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Duchenne Muscular Dystrophy (DMD) patients showed signs of disease progress slowing down, after oral consumption of Neu REFIX ß-glucan for 45 days along with routine medications.

Key Points: 
  • Duchenne Muscular Dystrophy (DMD) patients showed signs of disease progress slowing down, after oral consumption of Neu REFIX ß-glucan for 45 days along with routine medications.
  • View the full release here: https://www.businesswire.com/news/home/20230803923960/en/
    Duchenne Muscular Dystrophy (DMD); Progress of disease & gradual disability with aging, current therapies & Neu-REFIX Beta glucans' multipronged potentials, illustrated.
  • A rare genetic disease with approximately 5000 patients in Japan, 3000 in GCC, fewer than 50000 in USA.
  • Clinical research in Limb-Girdle Muscle Dystrophy (LGMD) , multiple sclerosis and psoriasis are underway, to evaluate Neu-REFIX’s efficacy as a universal immune modulator.

AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

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Thursday, August 3, 2023
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Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.

Key Points: 
  • Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.
  • "Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease," said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i.
  • "Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy.
  • The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life."

BridgeBio Pharma Announces Opportunity for Accelerated Approval Pathway in Limb-girdle Muscular Dystrophy Type 2I (LGMD2I/R9) Based on Glycosylated Alpha-dystroglycan (⍺DG) Levels and Announces First Patient Dosed in FORTIFY Phase 3 Study

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Monday, July 31, 2023
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BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418.

Key Points: 
  • BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418.
  • The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418.
  • FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval.
  • “Currently, our Phase 2 data suggest that glycosylated αDG levels are improved and sustained over time following treatment with BBP-418.

New 'Voice of the Patient' Report Highlights Unmet Medical Needs of Six Subtypes of Limb-Girdle Muscular Dystrophy

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Monday, May 15, 2023
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This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9.

Key Points: 
  • This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9.
  • An additional 136 patients and family members responded to a live survey and over 250 written comments were submitted.
  • The LGMD Coalition is appreciative for support from our sponsors, Sarepta Therapeutics, AskBio, Edgewise Therapeutics, ML BioSolutions, and Vita Therapeutics.
  • We believe that their engagement in this process provided insights into these subtypes of LGMD which will help the research community develop effective therapies."

Third International Limb Girdle Muscular Dystrophy Conference to Be Held in Washington, D.C. On October 27-29, 2023

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Wednesday, April 5, 2023
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LANCASTER, Pa., April 5, 2023 /PRNewswire/ -- On October 27, 2023, the third International Limb Girdle Muscular Dystrophy (LGMD) Conference will be hosted by The Speak Foundation at the Grand Hyatt Washington, DC.

Key Points: 
  • LANCASTER, Pa., April 5, 2023 /PRNewswire/ -- On October 27, 2023, the third International Limb Girdle Muscular Dystrophy (LGMD) Conference will be hosted by The Speak Foundation at the Grand Hyatt Washington, DC.
  • The second International LGMD conference was in 2021 and was offered in a virtual format due to COVID-19.
  • The conference will be back in person this year and will be offering live-streamed sessions for those unable to attend.
  • There will be a number of "breakout sessions" at the Conference geared toward providing practical advice to the patient community.

Sarepta Therapeutics Announces Initiation of VOYAGENE, a Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4

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Friday, February 17, 2023
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the first patient has been dosed in Study SRP-9003-102.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the first patient has been dosed in Study SRP-9003-102.
  • Also known as VOYAGENE, Study 9003-102 is a phase 1 study of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E).
  • VOYAGENE is a U.S.-only study enrolling ambulant patients aged 18 years or older and non-ambulant patients, ages 4-50 years, using clinical process SRP-9003 material.
  • “Sarepta has previously disclosed positive expression and function data from our initial clinical study, Study SRP-9003-101, exploring multiple doses of SRP-9003 in a younger, ambulant population.

Sarepta and Catalent Expand Strategic Manufacturing Partnership With Commercial Supply Agreement for Duchenne Muscular Dystrophy Gene Therapy Candidate

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Thursday, January 5, 2023
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The agreement also structures how Catalent may support multiple gene therapy candidates in Sarepta’s pipeline for limb-girdle muscular dystrophy (LGMD).

Key Points: 
  • The agreement also structures how Catalent may support multiple gene therapy candidates in Sarepta’s pipeline for limb-girdle muscular dystrophy (LGMD).
  • Under the terms of this expanded agreement, Catalent will be Sarepta’s primary commercial manufacturing partner for this therapy.
  • “Sarepta is working as quickly as possible to advance new genetic medicines to treat progressive neuromuscular diseases like Duchenne and LGMD.
  • Catalent is also the only contract development and manufacturing organization (CDMO) with a facility approved by the FDA for commercial manufacturing of an AAV gene therapy.

Nyheim Hines of the Buffalo Bills to Wear Cleats for Muscular Dystrophy Association on Game Day December 1

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Monday, November 28, 2022
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NEW YORK, Nov. 28, 2022 /PRNewswire/ -- For the sixth-consecutive season, players throughout the National Football League (NFL) will raise awareness for non-profit organizations and causes on the field through the My Cause My Cleats campaign, which allows players to utilize their platform by customizing their game day cleats to raise funds through auctions of the cleats. Players from across the NFL have supported the Muscular Dystrophy Association (MDA), and Nyheim Hines, 2022 MDA National Spokesperson, and running back for the Buffalo Bills will be wearing his cleats for MDA on game day, Thursday, December 1 for Buffalo vs New England. Watch Hines' My Cause My Cleats video posted on Instagram here. Hines is also the featured spokesperson in MDA's Public Service Announcement airing to promote the mission of the organization. Media can download PSAs here.

Key Points: 
  • Players from across the NFL have supported the Muscular Dystrophy Association (MDA), and Nyheim Hines, 2022 MDA National Spokesperson, and running back for the Buffalo Bills will be wearing his cleats for MDA on game day, Thursday, December 1 for Buffalo vs New England.
  • Hines is also the featured spokesperson in MDA's Public Service Announcement airing to promote the mission of the organization.
  • "Raising awareness for the Muscular Dystrophy Association is a priority for me off the field, because I have seen my mother, grandmother and uncle live with a neuromuscular disease," said Buffalo Bills running back Hines.
  • Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases.

Selecta Biosciences Reports Third Quarter 2022 Financial Results and Provides Business Update

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Thursday, November 3, 2022
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Selecta continues to work with its partners to develop a next generation IL-2 molecule to combine with ImmTOR and still anticipates selecting an IL-2 candidate by year-end 2022.

Key Points: 
  • Selecta continues to work with its partners to develop a next generation IL-2 molecule to combine with ImmTOR and still anticipates selecting an IL-2 candidate by year-end 2022.
  • In Q3 2022, Selecta received a $10 million milestone payment from Sobi following the completion of enrollment of DISSOLVE II in June 2022.
  • Selecta management will host a conference call at 8:30 AM ET today to provide a business update and review the companys third quarter 2022 financial results.
  • The Company specifically disclaims any intention to update any forward-looking statements included in this press release, except as required by law.