Muscular dystrophy

TransCode Therapeutics Announces Appointment of Daniel Vlock, M.D., as Chief Medical Officer

Retrieved on: 
Thursday, March 28, 2024
Transcoding, Internal medicine, Temple University Hospital, CFO, Oncology, Patient, RNA, University, Cancer, University of Pittsburgh, Yale School of Medicine, Baylor College of Medicine, Infection, Therapy, Harvard University, Interim, Muscular dystrophy, Yale University, Pharmacia, Pharmaceutical industry

BOSTON, March 28, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, announced today that Daniel Vlock, M.D., has been appointed as the company’s Chief Medical Officer.

Key Points: 
  • BOSTON, March 28, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, announced today that Daniel Vlock, M.D., has been appointed as the company’s Chief Medical Officer.
  • Dr. Vlock is a medical oncologist with over 25 years of industry experience and 15 years in academia, including renowned institutions Yale University, University of Pittsburgh, and Harvard University.
  • While at Pharmacia, Dr. Vlock ran the Celebrex Oncology program involving two pivotal clinical trials and over 3,500 patients.
  • Dr. Vlock initially will serve on a part-time basis through TransCode’s arrangement with BioBridges LLC, a life sciences consulting company.

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.

Celularity Submits Request to U.S. FDA for Orphan Drug Designation for PDA-002 Asset Treating Facioscapulohumeral Muscular Dystrophy

Retrieved on: 
Wednesday, March 20, 2024
CELU, Diagnosis, Shoulder, Biologics license application, Disease, Risk, Patient, Microsoft Exchange Server, Stem cell, IND, Facioscapulohumeral muscular dystrophy, Regenerative medicine, FDA, BLA, FSHD, Muscular dystrophy, Food, Pharmaceutical industry

FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).

Key Points: 
  • FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).
  • Orphan drug designation is a status given to drugs that show the potential to treat, prevent or diagnose serious or life-threatening diseases that affect fewer than 200,000 people in the U.S.
  • Earlier, Celularity received FDA Investigational New Drug Application clearance for PDA-002 in FSHD and plans to commence a phase 1/2 study in the second half of 2024.
  • The trial serves as an important component for submitting a Biologics License Application (BLA) to the FDA in the future as a potential treatment for FSHD and other types of muscular dystrophy.

Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne Muscular Dystrophy (DMD)

Retrieved on: 
Wednesday, March 13, 2024
Catalyst Pharmaceuticals, Steroid, Caregiver, Patient, Ageing, Duchenne muscular dystrophy, Hope, Fatality, Quality of life, Respiratory failure, Behavior, Vamorolone, FDA, DMD, Muscular dystrophy, Food, Pharmaceutical industry

CORAL GABLES, Fla., March 13, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company, today announced the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne Muscular Dystrophy ("DMD") in patients aged two years and older. Following the U.S. Food and Drug Administration ("FDA") approval on October 26, 2023, AGAMREE is now available by prescription and dispensed throughout the United States through a specialty pharmacy network.

Key Points: 
  • "Today, we proudly announced the U.S. commercial availability of AGAMREE, an innovative alternative steroid treatment for Duchenne Muscular Dystrophy.
  • Our Catalyst Pathways, a personalized program, is readily available to assist Duchenne Muscular Dystrophy patients and their families with the one‐on‐one education and financial support they need, thereby enhancing the access and affordability of AGAMREE for every patient.
  • For more information, caregivers and healthcare professionals can call 1-833-422-8259 or visit the Catalyst Pathways® website at www.yourcatalystpathways.com .
  • Duchenne Muscular Dystrophy, DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality.

NFL Running Back Nyheim Hines Supports Nation's Largest St. Patrick’s Day Fundraising Campaign to Benefit Muscular Dystrophy Association

Retrieved on: 
Wednesday, March 13, 2024
Cleveland Browns, Nyheim Hines, ALS, MDA, Partnership, Muscular dystrophy, Research, Muscular Dystrophy Association, Family, NFL, Neuromuscular disease

Hines proudly wears the 2024 limited edition MDA Shamrocks t-shirt designed by MDA Board member Matthew Plummer who lives with neuromuscular disease.

Key Points: 
  • Hines proudly wears the 2024 limited edition MDA Shamrocks t-shirt designed by MDA Board member Matthew Plummer who lives with neuromuscular disease.
  • With family members who live or have lived with muscular dystrophy, Hines continues to generate awareness and raise critical funds for the neuromuscular disease community.
  • "It's truly humbling to continue to raise awareness for the neuromuscular disease community through the mission of Muscular Dystrophy Association.
  • It’s our privilege to collaborate with Nyheim to bring the fruits of our mission to his family and millions more across the country."

Bausch + Lomb Will Present New Scientific Data and Analyses at the American Society of Cataract and Refractive Surgery Annual Meeting

Retrieved on: 
Wednesday, March 27, 2024
Science, Other Science, Research, Pharmaceutical, Optical, Surgery, General Health, Health, Momentum, YAG, Toric, Failure, IOP, Retinal, Glaucoma, Exercise, DSM-IV codes, Neuropathic pain, ASCRS, History, Sale, Contact, Dry, Refraction, Bausch & Lomb, Eye, System, Keratoconus, Medication discontinuation, Pain, Corneal topography, Telecanthus, Astigmatism, Aphakia, Correction, Uveitis, Surgeon, Diabetes, Risk, Eye injury, CTK, Retinal detachment, Side, Trial of the century, Visual acuity, Diagnosis, Safety, Contraindication, Emmetropia, Vision, Taste, Dry eye syndrome, Intraocular lens, Presbyopia, Real, Floater, Magnetic resonance, Contrast (vision), Human, GLARE, Infection, Use, FDA, Ranibizumab, Muscular dystrophy, Sickle cell disease, Refractive surgery, Retina, Direction, IDE, Allergy, User, Society, Bleeding, Autoimmune disease

In addition to these scientific presentations, the company will also sponsor educational events focused on these and other Bausch + Lomb products.

Key Points: 
  • In addition to these scientific presentations, the company will also sponsor educational events focused on these and other Bausch + Lomb products.
  • Bausch + Lomb activities at ASCRS:
    “Assessment of the Spectacle Independence and Visual Outcomes Following Bilateral Implantation of a Novel Toric IOL* Among Cataract Patients.” Harasymowycz et al.
  • “Outcomes of an Ultra-Low Cylinder Powered (0.90 D) Toric versus Non-Toric IOL in Low Astigmatic Patients Undergoing Cataract Surgery.” Muzychuk et al.
  • “Quality of Vision and Clinical Outcomes of a Novel Trifocal IOL* Implanted Bilaterally After Cataract Surgery: A Canadian Multicenter Study.” Harasymowycz et al.

Satellos Bioscience Announces 2023 Year End Financial Results and Operational Highlights

Retrieved on: 
Wednesday, March 27, 2024
Biotechnology, Health, Clinical Trials, Research, Conference, TSX Venture Exchange, MSCL, Regeneration, CFO, G&A, Toronto Stock Exchange, Muscular dystrophy, Doctor of Philosophy, MD, Senior, MBA, GMP, IND, Bank statement, TSX, Regenerative medicine, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Investment, Chemistry, Toxicology, Therapy, AAK1, OTCQB, FDA, GLP, Animal, DMD, FSHD, Hand, Episcopal conference, Food, CPA, Pharmaceutical industry

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the year ended December 31, 2023.

Key Points: 
  • Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the year ended December 31, 2023.
  • Subsequent to the year end, on March 4, 2024, Satellos announced positive preclinical data presented at the Muscular Dystrophy Association Clinical and Scientific Conference.
  • Satellos also announced that SAT-3247 would be nominated as its lead development candidate with SAT-3153 becoming the backup development candidate.
  • Satellos’ audited financial statements for the year ended December 31, 2023, and the related management’s discussion and analysis (MD&A) will be available on SEDAR+ at www.sedarplus.ca .

Atamyo Therapeutics Obtains Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-200, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5

Retrieved on: 
Tuesday, March 26, 2024
Health, Genetics, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, CNRS, Neurology, AAV, Motivation, Environment, Scientific method, Patient, CTA, DSM-IV codes, Biomarker, Research, ANSM, University, EMA, European Medicines Agency, Clinical trial, Therapy, Muscular dystrophy, Safety, Pathology, Weakness, Cardiomyopathy, Pharmaceutical industry

This authorization was first granted by the Italian Medicines Agency (AIFA), then by the French Medicines Agency (ANSM).

Key Points: 
  • This authorization was first granted by the Italian Medicines Agency (AIFA), then by the French Medicines Agency (ANSM).
  • Giacomo Comi, Full Professor of Neurology at the University of Milan (Italy), and principal investigator of this trial.
  • ATA-200, a gene therapy candidate for LGMD2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan protein.
  • In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein.

Scholar Rock Reports Full Year 2023 Financial Results and Highlights Business Progress

Retrieved on: 
Tuesday, March 19, 2024
Research, FDA, Clinical Trials, Cardiology, Biotechnology, Pharmaceutical, Health, Science, Oncology, Other Science, Overweight, SMN, Conference, Obesity, Keystone, MDA, Gilead Sciences, SAPPHIRE, SMA, Manuscript, Myostatin, Rock, Muscular dystrophy, Muscular Dystrophy Association, Trial of the century, Efficacy, Clinical trial, Therapy, Literature, Lunch, Pharmaceutical industry

“In 2023, Scholar Rock made significant progress across our industry-leading antimyostatin pipeline, and we are poised for a transformational year ahead.

Key Points: 
  • “In 2023, Scholar Rock made significant progress across our industry-leading antimyostatin pipeline, and we are poised for a transformational year ahead.
  • Scholar Rock remains on track to report topline data from the Phase 3 trial in the fourth quarter of 2024.
  • Scholar Rock also presented a literature review that describes the ongoing burden and significant unmet needs in SMA despite advancements in treatment.
  • Dr. Qatanani joined Scholar Rock in 2021 and has been leading all research functions since 2022 and driving Scholar Rock’s early-stage pipeline, including SRK-439.