Limb-girdle muscular dystrophy

Insights on the Limb-Girdle Muscular Dystrophy Global Market to 2024 - Synthesize Insights for Business Development & Licensing - ResearchAndMarkets.com

Retrieved on: 
Monday, July 13, 2020
Health, Pharmaceutical, Muscular dystrophy, Organ systems, Limb-girdle muscular dystrophy, Medicine, Dystrophy, Stamulumab, Limb girdle syndrome

This report provides comprehensive insights into the Limb-Girdle Muscular Dystrophy pipeline, epidemiology, market valuations, drug sales, market forecast, drug forecasts, and market shares.

Key Points: 
  • This report provides comprehensive insights into the Limb-Girdle Muscular Dystrophy pipeline, epidemiology, market valuations, drug sales, market forecast, drug forecasts, and market shares.
  • This research analyzes and forecasts the Limb-Girdle Muscular Dystrophy market size and drug sales.
  • 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, and phase 1, 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 2019 by countries.

Limb Girdle Muscular Dystrophy (LGMD) Market Study 2020: Clinical Development Scenario, Growth Prospects and Pipeline Landscape - ResearchAndMarkets.com

Retrieved on: 
Friday, July 3, 2020
Pharmaceutical, Health, Clinical trials, Muscular dystrophy, Limb-girdle muscular dystrophy, Dystrophy, Medicine, Branches of biology, Proteins, Health

Limb Girdle Muscular Dystrophy (LGMD) Pipeline Insight, 2020 report outlays comprehensive insights of present clinical development scenario and growth prospects across the Limb Girdle Muscular Dystrophy (LGMD) market.

Key Points: 
  • Limb Girdle Muscular Dystrophy (LGMD) Pipeline Insight, 2020 report outlays comprehensive insights of present clinical development scenario and growth prospects across the Limb Girdle Muscular Dystrophy (LGMD) market.
  • A detailed picture of the Limb Girdle Muscular Dystrophy (LGMD) pipeline landscape is provided, which includes the disease overview and Limb Girdle Muscular Dystrophy (LGMD) treatment guidelines.
  • The assessment part of the report embraces in-depth Limb Girdle Muscular Dystrophy (LGMD) commercial assessment and clinical assessment of the Limb Girdle Muscular Dystrophy (LGMD) pipeline products from the pre-clinical developmental phase to the marketed phase.
  • The Limb Girdle Muscular Dystrophy (LGMD) report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Limb Girdle Muscular Dystrophy (LGMD) across the complete product development cycle, including all clinical and nonclinical stages.

Sarepta Therapeutics Signs Agreement with Hansa Biopharma for Imlifidase

Retrieved on: 
Thursday, July 2, 2020
Branches of biology, Life sciences, Biology, Muscular dystrophy, RTT, Biotechnology, Corvallis, Oregon, Sarepta Therapeutics, Applied genetics, Duchenne muscular dystrophy, Limb-girdle muscular dystrophy, Gene therapy

CAMBRIDGE, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an agreement with Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare Immunoglobulin G (IgG) mediated diseases, for imlifidase.

Key Points: 
  • CAMBRIDGE, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an agreement with Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare Immunoglobulin G (IgG) mediated diseases, for imlifidase.
  • Under the terms of the agreement, Sarepta obtains an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy administration in Duchenne muscular dystrophy (DMD) and Limb-girdlemuscular dystrophy (LGMD), for patients who may otherwise not be eligible for treatment.
  • Hansa will book all sales of imlifidase and will earn tiered royalties up to the mid-teens on any incremental gene therapy sales that arise from treating antibody-positive patients enabled through imlifidase pre-treatment.
  • For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta.

BridgeBio Pharma’s ML Bio Solutions Announces Dosing of First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)

Retrieved on: 
Thursday, June 11, 2020
Fukutin-related protein, Fukutin, Limb-girdle muscular dystrophy, Muscular dystrophy

The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics and food effect of BBP-418 in healthy volunteers.

Key Points: 
  • The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics and food effect of BBP-418 in healthy volunteers.
  • LGMD2i is an inherited muscular dystrophy that is associated with two mutant copies of a gene called fukutin-related protein (FKRP).
  • The potential of a disease-modifying treatment from ML Bio Solutions for LGMD2i represents a beacon of hope for our community.
  • Moreover, ML Bio Solutions operates in a very competitive and rapidly changing environment in which new risks emerge from time to time.

Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E

Retrieved on: 
Monday, June 8, 2020
Muscular dystrophy, RTT, Medicine, Dystrophy, Duchenne muscular dystrophy, Duchenne, Limb-girdle muscular dystrophy, Branches of biology, Health

SRP-9003 employs the same vector, AAVrh74, and same promoter, MHCK7, as SRP-9001, our therapy in development to treat Duchenne muscular dystrophy.

Key Points: 
  • SRP-9003 employs the same vector, AAVrh74, and same promoter, MHCK7, as SRP-9001, our therapy in development to treat Duchenne muscular dystrophy.
  • Sarepta previously shared data from Cohort 1 in 2019, including positive and robust expression and biomarker data and positive 9-month functional results .
  • Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10.
  • For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review theSECfilings made by Sarepta.

BridgeBio Pharma Reports Third Quarter 2019 Financial Results and Highlights Portfolio Progress

Retrieved on: 
Thursday, November 7, 2019
Muscular dystrophy, RTT, Limb-girdle muscular dystrophy, Cash and cash equivalents, Neuromuscular disease, Dystrophy, Medicine, Health

Ended quarter with $446.1 million in cash, cash equivalents and marketable securities, excluding Eidos

Key Points: 
  • Ended quarter with $446.1 million in cash, cash equivalents and marketable securities, excluding Eidos
    PALO ALTO, Calif., Nov. 07, 2019 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ: BBIO) today reported financial results for the third quarter, which ended September 30, 2019, and provided recent highlights across the companys research and development portfolio.
  • We are growing our pipeline as well, adding BBP-418, a novel treatment for the neuromuscular disorder limb-girdle muscular dystrophy type 2i.
  • We remain on track to put five or more programs into the clinic next year on a risk-adjusted basis.
  • Third Quarter 2019 Financial Results:

Catabasis Pharmaceuticals and the Jain Foundation Announce a Preclinical Research Collaboration to Study Edasalonexent in Dysferlinopathy

Retrieved on: 
Thursday, September 26, 2019
Science, Biotechnology, Pharmaceutical, Research, Health, Clinical trials, Proteins, Dysferlin, Muscular dystrophy, Catabasis Pharmaceuticals, Ajit Jain, Limb-girdle muscular dystrophy

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and the Jain Foundation , a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, announced a preclinical research collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy.

Key Points: 
  • Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and the Jain Foundation , a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, announced a preclinical research collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy.
  • Edasalonexent, an oral small molecule designed to inhibit NF-kB, has the potential to slow disease progression in dysferlin-deficient populations.
  • Under this collaboration, Catabasis and the Jain Foundation are conducting a preclinical study to evaluate the potential of edasalonexent as a therapeutic intervention for Dysferlinopathy by measuring disease progression in dysferlin-deficient mice treated with edasalonexent.
  • We look forward to working with Catabasis to advance research for Dysferlinopathy, said Laura Rufibach, Ph.D., and Doug Albrecht, Ph.D., Co-Presidents of the Jain Foundation.

Sarepta Therapeutics to Announce 9-Month Functional Results from the SRP-9003 Gene Therapy Trial to Treat Limb-girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy

Retrieved on: 
Wednesday, September 25, 2019
Branches of biology, Articles, Life sciences, Limb-girdle muscular dystrophy, Gene delivery, Applied genetics, Biotechnology, Therapy, Muscular dystrophy, Gene therapy

Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

Key Points: 
  • Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
  • The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers.
  • Please specify to the operator that you would like to join the "Sarepta hosted LGMD results call."
  • The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing.

BridgeBio Pharma expands its research into neuromuscular disease to include Limb-Girdle Muscular Dystrophy Type 2 (LGMD2i) with new subsidiary ML Bio Solutions

Retrieved on: 
Thursday, September 5, 2019
Muscular dystrophy, Organ systems, Branches of biology, RTT, Medicine, Limb-girdle muscular dystrophy, Dystroglycan, Dystrophy, Fukuyama congenital muscular dystrophy, Congenital muscular dystrophy

The McColl Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health will continue to support the program alongside BridgeBio.

Key Points: 
  • The McColl Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health will continue to support the program alongside BridgeBio.
  • LGMD2i is an autosomal recessive disease wherein loss-of-function mutations in the FKRP gene lead to decreased glycosylation of alpha-dystroglycan (-DG).
  • LGMD2i is a progressive and debilitating disease that affects an estimated 7,000 patients across the US and EU, with more affected globally.
  • ML Bio Solutions, a subsidiary of BridgeBio Pharma based in Charlotte, NC, is a biotechnology company focused on developing a small molecule as an oral substrate supplementation therapy for LGMD2i.

Limb-Girdle Muscular Dystrophy Clinical Trial Pipeline Highlights 2019 - Global Forecast to 2024 by Clinical Trial Stage, Drug Mechanism Class, Company - ResearchAndMarkets.com

Retrieved on: 
Friday, June 14, 2019
Health, Clinical trials, Pharmaceutical, Health, Organ systems, Medicine, Muscular dystrophy, RTT, Limb-girdle muscular dystrophy, Dystrophy, Clinical trial, Draft:CureDuchenne, Catabasis Pharmaceuticals

The "Global Limb-Girdle Muscular Dystrophy Clinical Trial Pipeline Highlights - 2019" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Limb-Girdle Muscular Dystrophy Clinical Trial Pipeline Highlights - 2019" report has been added to ResearchAndMarkets.com's offering.
  • Limb-Girdle Muscular Dystrophy Pipeline Highlights - 2019 provides most up-to-date information on key pipeline products in the global Limb-Girdle Muscular Dystrophy market.
  • It covers emerging therapies for Limb-Girdle Muscular Dystrophy in active clinical development stages including early and late stage clinical trials.
  • The report provides Limb-Girdle Muscular Dystrophy pipeline products by clinical trial stages including both early and late stage development - phase 3 clinical trials, phase 2 clinical trials, phase 1 clinical trials, preclinical research, and discovery stage.