Limb–girdle muscular dystrophy

Limb-Girdle Muscular Dystrophy Leaders Advance Collaborative Dialogue to Accelerate Drug Development to Address the Urgent Unmet Need in Limb-Girdle Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024

The meeting focused on six LGMD subtypes: 2A/R1, 2B/R2, 2C/R5, 2D/R3, 2E/R4, and 2I/R9.

Key Points: 
  • The meeting focused on six LGMD subtypes: 2A/R1, 2B/R2, 2C/R5, 2D/R3, 2E/R4, and 2I/R9.
  • We are thrilled that the workshop advanced a collaborative dialogue and brought people together from every facet of the drug development process to drive progress for patients.
  • This was truly a monumental event for the LGMD community and a model for others to follow in the future."
  • In-depth documentation of the workshop's findings and next steps is in progress and will be shared when available.

Limb-Girdle Muscular Dystrophy Scientific Leaders - Together With the FDA - Will Come Together for a Drug Development Workshop on February 8, 2024

Retrieved on: 
Wednesday, December 13, 2023

ROCKVILLE, Md., Dec. 13, 2023 /PRNewswire/ -- On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a multi-stakeholder LGMD Scientific Drug Development Workshop in Rockville, Maryland.

Key Points: 
  • ROCKVILLE, Md., Dec. 13, 2023 /PRNewswire/ -- On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a multi-stakeholder LGMD Scientific Drug Development Workshop in Rockville, Maryland.
  • The workshop will continue to drive the momentum for the sense of urgency needed by the patient community to accelerate LGMD drug development.
  • "This workshop is a continuing step to work with the FDA and LGMD scientific leaders on pushing critical drug development forward.
  • The LGMD Scientific Workshop will be a focused discussion with clinicians, scientific researchers, drug developers, regulators, patients and patient advocates on the characteristics of LGMD that drive drug development programs.

Vita Therapeutics Closes $31 Million Series B Financing to Develop Cell Therapies for Neuromuscular Diseases and Cancers

Retrieved on: 
Wednesday, October 12, 2022

Vita Therapeutics , a cell engineering company harnessing the power of genetics to develop novel cellular therapies to treat muscular dystrophies and cancers, today announced the completion of a $31 million Series B financing.

Key Points: 
  • Vita Therapeutics , a cell engineering company harnessing the power of genetics to develop novel cellular therapies to treat muscular dystrophies and cancers, today announced the completion of a $31 million Series B financing.
  • Vita Therapeutics current pipeline includes lead program, VTA-100 for the treatment of LGMD2A, VTA-120 for the treatment of FSHD, and VTA-300 targeting multiple cancers.
  • Vita Therapeutics is a biotechnology company developing state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers.
  • Vita Therapeutics uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients.

Registration Now Open for 2023 MDA Clinical & Scientific Conference, to Showcase Cutting Edge Research Advancements and Clinical Achievements in Neuromuscular Disease

Retrieved on: 
Monday, September 12, 2022

NEW YORK, Sept. 12, 2022 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) today announced that registration is open for the 2023 MDA Clinical & Scientific Conference being held March 19-22, 2023. MDA is the largest worldwide convener of the neuromuscular disease community of renowned researchers, clinicians, academicians, advocates, and industry leaders both in person at the Hilton Anatole in Dallas, Texas and via live stream.

Key Points: 
  • What is equally important is that we convene MDA's Care Center Network of neuromuscular disease specialists, connecting them to the latest clinical trials and FDA approved treatments.
  • They are also developing new treatments and creating the pipeline of progress that is the hallmark of today's neuromuscular disease frontier."
  • Before the conference, MDA will once again host the annual gathering of the Neuromuscular Advocacy Collaborative (NMAC).
  • For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families.

AskBio CEO and Co-Founder Sheila Mikhail receives 2022 Entrepreneurial Award from University of Chicago Booth School of Business

Retrieved on: 
Friday, June 3, 2022

RESEARCH TRIANGLE PARK, N.C., June 3, 2022 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), an independently operated subsidiary of Bayer AG, today announced that CEO and Co-Founder Sheila Mikhail was recognized by the University of Chicago Booth School of Business for outstanding professional achievement among its alumni. As a 1993 finance MBA graduate with honors, Ms. Mikhail has been recognized with the 2022 Entrepreneurial Award.

Key Points: 
  • As a 1993 finance MBA graduate with honors, Ms. Mikhail has been recognized with the 2022 Entrepreneurial Award.
  • "This award is tremendously meaningful as the Booth School of Business is one of the nation's top ranked business schools," said AskBio Co-Founder and CEO Sheila Mikhail.
  • Ms. Mikhail earned a JD with honors from Northwestern University, a finance MBA with honors from the University of Chicago, and a BS with highest honors from the University of Illinois at Urbana-Champaign.
  • "The recognition by University of Chicago Booth School of Business is a testament of her unparalleled business acumen combined with her significant command of genetic science"
    The Distinguished Alumni awards are only available to graduates of Chicago Booth.

Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape Report 2021: Pipeline, Epidemiology, Market Valuations, Drug Sales, Drug Forecasts, and Market Shares 2018-2026 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, November 2, 2021

The "Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2021" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2021" report has been added to ResearchAndMarkets.com's offering.
  • Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2021, provides comprehensive insights into Limb-Girdle Muscular Dystrophy pipeline, epidemiology, market valuations, drug sales, market forecast, drug forecasts, and market shares.
  • This research covers the following - Limb-Girdle Muscular Dystrophy treatment options, Limb-Girdle Muscular Dystrophy late-stage clinical trials pipeline, Limb-Girdle Muscular Dystrophy prevalence by countries, Limb-Girdle Muscular Dystrophy market size and forecast by countries, key market events and trends, drug sales and forecast by countries, and market shares by countries.
  • Limb-Girdle Muscular Dystrophy pipeline: Find out drugs in clinical trials for the treatment of Limb-Girdle Muscular Dystrophy by development phase 3, phase 2, by pharmacological class and company
    Limb-Girdle Muscular Dystrophy drugs: Identify key drugs marketed and prescribed for Limb-Girdle Muscular Dystrophy in the US, including trade name, molecule name, and company
    Limb-Girdle Muscular Dystrophy drugs sales: Find out the sales value for Limb-Girdle Muscular Dystrophy drugs by countries
    Limb-Girdle Muscular Dystrophy market valuations: Find out the market size for Limb-Girdle Muscular Dystrophy drugs in 2020 by countries.

Global Limb-Girdle Muscular Dystrophy Market Report 2021: Pipeline, Epidemiology, Valuations, Sales, Forecasts, and Market Shares 2018-2026 - ResearchAndMarkets.com

Retrieved on: 
Friday, September 17, 2021

The "Global Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2021" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2021" report has been added to ResearchAndMarkets.com's offering.
  • Global Limb-Girdle Muscular Dystrophy Market and Competitive Landscape - 2021, provides comprehensive insights into Limb-Girdle Muscular Dystrophy pipeline, epidemiology, market valuations, drug sales, market forecast, drug forecasts, and market shares.
  • This research covers the following - Limb-Girdle Muscular Dystrophy treatment options, Limb-Girdle Muscular Dystrophy late-stage clinical trials pipeline, Limb-Girdle Muscular Dystrophy prevalence by countries, Limb-Girdle Muscular Dystrophy market size and forecast by countries, key market events and trends, drug sales and forecast by countries, and market shares by countries.
  • Limb-Girdle Muscular Dystrophy pipeline: Find out drugs in clinical trials for the treatment of Limb-Girdle Muscular Dystrophy by development phase 3, phase 2, by pharmacological class and company
    Limb-Girdle Muscular Dystrophy drugs: Identify key drugs marketed and prescribed for Limb-Girdle Muscular Dystrophy in the US, including trade name, molecule name, and company
    Limb-Girdle Muscular Dystrophy drugs sales: Find out the sales value for Limb-Girdle Muscular Dystrophy drugs by countries
    Limb-Girdle Muscular Dystrophy market valuations: Find out the market size for Limb-Girdle Muscular Dystrophy drugs in 2020 by countries.

AskBio Announces IND for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Cleared to Proceed by U.S. FDA

Retrieved on: 
Tuesday, May 25, 2021

LION-101 is a novel recombinant adeno-associated virus (rAAV) based vector being developed as a one-time intravenous infusion for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).

Key Points: 
  • LION-101 is a novel recombinant adeno-associated virus (rAAV) based vector being developed as a one-time intravenous infusion for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).
  • AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library.
  • Limb-girdle muscular dystrophy: MedlinePlus Genetics
    2 National Institutes of Health -- National Center of Advancing Translational Sciences: Genetic and Rare Diseases Information Center.
  • Limb-girdle muscular dystrophy type 2I | Genetic and Rare Diseases Information Center (GARD) an NCATS Program (nih.gov)
    View original content to download multimedia: http://www.prnewswire.com/news-releases/askbio-announces-ind-for-lion-10...

Sarepta Therapeutics to Share Expression and Safety Results from Study 103 (ENDEAVOR) Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Monday, May 17, 2021

Study 103 is the first clinical trial using Sarepta\xe2\x80\x99s commercially representative material for SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for the treatment of Duchenne muscular dystrophy.

Key Points: 
  • Study 103 is the first clinical trial using Sarepta\xe2\x80\x99s commercially representative material for SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for the treatment of Duchenne muscular dystrophy.
  • The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers.
  • We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.
  • Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

Global Limb-Girdle Muscular Dystrophy Markets, Epidemiology and Patient Flow Analysis Report 2021 - ResearchAndMarkets.com

Retrieved on: 
Friday, May 14, 2021

b'The "Global Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow Analysis - 2021, provides Limb-Girdle Muscular Dystrophy epidemiology, demographics, and patient flow.

Key Points: 
  • b'The "Global Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Limb-Girdle Muscular Dystrophy Epidemiology and Patient Flow Analysis - 2021, provides Limb-Girdle Muscular Dystrophy epidemiology, demographics, and patient flow.
  • The data is presented by Pharma G7 countries including the US, France, Spain, Italy, UK, Japan.\nThe research provides population data to characterize Limb-Girdle Muscular Dystrophy patients, history of the disease at the population level (Limb-Girdle Muscular Dystrophy prevalence, Limb-Girdle Muscular Dystrophy incidence) and at the clinical level (from diagnosis to treated patients).
  • It also helps to identify patient sub-groups (age, gender, sub-groups) to understand targeted population for research and development, commercialization.\nLimb-Girdle Muscular Dystrophy patient flow: Limb-Girdle Muscular Dystrophy prevalence, diagnosed, and drug-treated patients\nDemographics: Limb-Girdle Muscular Dystrophy patients by age group, gender\nThe data from this research will help executives:\nEstablish basis for Limb-Girdle Muscular Dystrophy market sizing, assessing market potential, and developing drug forecast models\nIdentify Limb-Girdle Muscular Dystrophy patients segments through age groups, gender, and disease sub-types\nEvaluate Limb-Girdle Muscular Dystrophy market opportunities, identify target patient population\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210514005192/en/\n'