Duchenne muscular dystrophy

Biophytis announces its 2023 financial results and provides an update on its business activities

Retrieved on: 
Wednesday, April 10, 2024
Annual, Notifiable disease, Annual report, Conference, Obesity, Euronext Growth, Goldman Sachs, Disease, MDA, Partnership, Risk, COVID-19, Patient, Duchenne muscular dystrophy, Sarcopenia, University, Respiratory failure, Clinical trial, Therapy, FDA, Pharmaceutical industry

2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.

Key Points: 
  • 2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.
  • Promising preclinical results for BIO101 (20-hydroxyecdysone) in obesity, suggesting beneficial metabolic effects on muscle and fat mass.
  • On this basis, Biophytis plans to start a phase 1/2 clinical trial in 2024, depending on its financial resources.
  • There is therefore significant doubt about the Company's ability to continue its business activities.

Biophytis announces its 2023 financial results and provides an update on its business activities

Retrieved on: 
Wednesday, April 10, 2024
Annual, Notifiable disease, Annual report, Conference, Obesity, Euronext Growth, Goldman Sachs, Disease, MDA, Partnership, Risk, COVID-19, Patient, Duchenne muscular dystrophy, Sarcopenia, University, Respiratory failure, Clinical trial, Therapy, FDA, Pharmaceutical industry

2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.

Key Points: 
  • 2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.
  • Promising preclinical results for BIO101 (20-hydroxyecdysone) in obesity, suggesting beneficial metabolic effects on muscle and fat mass.
  • On this basis, Biophytis plans to start a phase 1/2 clinical trial in 2024, depending on its financial resources.
  • There is therefore significant doubt about the Company's ability to continue its business activities.

Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Retrieved on: 
Monday, April 1, 2024
Patient, DNA, Hope, Tropism, Duchenne muscular dystrophy, Duchenne, NOS1, Safety, FDA, DMD, Food, Pharmaceutical industry

CHARLESTOWN, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.

Key Points: 
  • “Solid’s receipt of Rare Pediatric Disease Designation for SGT-003 highlights the continuing need for transformational treatments for this devastating disease,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.
  • “The key components of SGT-003 were rationally designed to improve on first generation gene therapies to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes.
  • nNOS is believed to play a crucial role in both muscular function and endurance,” said Dr. Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences.
  • “We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need.”

Santhera Announces NDA for Vamorolone in Duchenne Muscular Dystrophy Accepted and Granted Priority Review by China’s NMPA

Retrieved on: 
Wednesday, March 27, 2024
Diagnosis, NMPA, Patient, Ageing, NDA, Breakthrough, CDE, Duchenne muscular dystrophy, Therapy, Vamorolone, Safety, File, DMD, Neuromuscular disease, Pharmaceutical industry

Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China.

Key Points: 
  • Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China.
  • The Center for Drug Evaluation (CDE) of the Chinese drug authority NMPA accepted the filing and granted priority review for vamorolone in DMD for patients aged 4 years and older which could, subject to a positive outcome, lead to approval by Q1 2025.
  • Duchenne muscular dystrophy is a rare neuromuscular disease affecting about 70,000 patients in China.
  • The submission is further supported by a study which investigated the pharmacokinetic parameters of vamorolone in healthy adult Chinese volunteers.

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Civil service commission, Wasting, Diagnosis, Atrophy, Mutation, B cell, Weakness, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Death, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, Safety, European Commission, Muscle weakness, Marketing, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Retrieved on: 
Tuesday, April 9, 2024
Community, Eurostat, Civil service commission, Diagnosis, U1 spliceosomal RNA, Capsid, Gene, B cell, Cell, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Protein, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, European Commission, Safety, Muscle weakness, Blood, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Burn Boot Camp and Muscular Dystrophy Association Team Up for 8th Annual 'Be Their Muscle' Philanthropic Event

Retrieved on: 
Monday, March 25, 2024
Research, Employment, Entrepreneurship, Skeletal muscle, MDA, ALS, Partnership, Air travel, Education, Duke University Hospital, Social media, Muscular dystrophy, Muscular Dystrophy Association, Senior, Empowerment, Duchenne muscular dystrophy, Duchenne, Clinical trial, FDA, Summer camp, Pharmaceutical industry

Charlotte, North Carolina, March 25, 2024 (GLOBE NEWSWIRE) -- Burn Boot Camp, a leading boutique fitness franchise, has teamed up with the Muscular Dystrophy Association (MDA) for its eighth annual national 'Be Their Muscle' philanthropic event.

Key Points: 
  • Charlotte, North Carolina, March 25, 2024 (GLOBE NEWSWIRE) -- Burn Boot Camp, a leading boutique fitness franchise, has teamed up with the Muscular Dystrophy Association (MDA) for its eighth annual national 'Be Their Muscle' philanthropic event.
  • Muscular Dystrophy Association is one of Burn Boot Camp's longest-standing national philanthropic partners," explained Morgan Kline, CEO and Co-Founder of Burn Boot Camp.
  • "Here at the Muscular Dystrophy Association, we stand hand in hand with Burn Boot Camp in our shared commitment to empower communities nationwide.
  • MDA and Burn Boot Camp will be posting throughout the campaign on social media using @MDAorg and @burnbootcamp with #BeTheirMuscle.

Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, March 21, 2024
HDAC, Gene expression, Cell, Disease, MDA, Associate, Patient, Research, DNA, Public policy, University, Muscular dystrophy, Doctor of Philosophy, Muscular Dystrophy Association, Duchenne muscular dystrophy, Clinical trial, Therapy, Histone deacetylase inhibitor, Stair climbing, Plasma protein binding, FDA, Steroid, DMD, Parent, Lancet, Pediatrics, Human, Food, Pharmaceutical industry

"The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA.

Key Points: 
  • "The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA.
  • “Muscular Dystrophy Association’s funding was fundamental for the discovery of HDACi in the treatment of Duchenne muscular dystrophy.
  • “We’re excited to celebrate this additional treatment option for people living with Duchenne muscular dystrophy.
  • My son is an example,” said Jessica Curran, MDA family member and mother of Conner who lives with Duchenne muscular dystrophy.

Capricor Therapeutics to Present at Upcoming Investor Conferences

Retrieved on: 
Thursday, March 21, 2024
Therapy, SAN, Duchenne muscular dystrophy, Biotechnology, DMD, Exosome

SAN DIEGO, March 21, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, today announced that the Company is scheduled to present at two upcoming investor conferences.

Key Points: 
  • SAN DIEGO, March 21, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, today announced that the Company is scheduled to present at two upcoming investor conferences.
  • Dr. Linda Marbán, Capricor’s chief executive officer will present updates on Capricor’s recent progress on its lead program of CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD) as well as other general scientific and corporate updates.
  • For more information on these presentations, please visit Capricor's website at http://capricor.com/news/events/ .

Santhera’s Partner Catalyst Pharmaceuticals Launches AGAMREE® (Vamorolone) in the United States

Retrieved on: 
Thursday, March 14, 2024
Catalyst Pharmaceuticals, Patient, Duchenne muscular dystrophy, Vamorolone, Food, FDA, Catalysis, DMD, Fine chemical

AGAMREE is the first DMD treatment approved across the U.S., EU and UK

Key Points: 
  • AGAMREE is the first DMD treatment approved across the U.S., EU and UK
    Pratteln, Switzerland, March 14, 2024 – Santhera Pharmaceuticals (SIX: SANN) is pleased to note the launch of AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in the United States (U.S.) by Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX), the Company’s commercialization partner for North America.
  • “Congratulations to our partner Catalyst on the launch of AGAMREE® in the United States.
  • According to the license agreement between the companies, first announced in June 2023 , Catalyst holds an exclusive North American license to commercialize AGAMREE for DMD and all potential future indications.
  • Under the agreement, Catalyst will pay Santhera sales-based milestones of up to USD 105 million as well as up to low-teen percentage royalties and will assume Santhera's corresponding third-party royalty obligations on AGAMREE sales in all indications in North America.