Muscle weakness

Selecta Biosciences Announces Merger with Cartesian Therapeutics

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Lunedì, Novembre 13, 2023
Acquisition, DNA, Entrepreneurship, CVR College of Engineering, Patient, Autoimmune disease, SVB Securities, Lancet Neurology, Webcast, CVR, Food, PIPE, Toxicity, Covington & Burling, Transfection, RNA, GMP, Therapy, Risk, Myasthenia gravis, AAAD, Pharmacokinetics, Mergers and acquisitions, Orphan, Autoimmunity, The Lancet, LLP, Neutrophil extracellular traps, Fatigue, Lupus, Muscle weakness, Event, Vaccine, Mobile phone, Pharmaceutical industry, Management, Security (finance), Selecta, Cartesian, Quality

and GAITHERSBURG, Md., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) (the Company) today announced that it has merged with Cartesian Therapeutics, Inc., a clinical-stage biotechnology company pioneering RNA cell therapies for autoimmune diseases.

Key Points: 
  • and GAITHERSBURG, Md., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) (the Company) today announced that it has merged with Cartesian Therapeutics, Inc., a clinical-stage biotechnology company pioneering RNA cell therapies for autoimmune diseases.
  • In connection with the merger, Selecta announced a $60.25 million private financing led by Timothy A. Springer, Ph.D., member of the Selecta Board of Directors.
  • Concurrent with the merger, the combined company has been renamed Cartesian Therapeutics, Inc.
  • Selecta and Cartesian will host a conference call today, Monday, November 13, 2023, at 9:00 am ET to discuss the merger.

Rocket Pharmaceuticals Appoints R. Keith Woods to Board of Directors

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Martedì, Dicembre 12, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Efgartigimod alfa, Senior, Disorder, Florida State University, Hematology, Patient, Eisai, Alexion Pharmaceuticals, Marketing, FA, Muscle weakness, Roche, R, Fanconi anemia, Medication, Amgen, Pharmaceutical industry, Management, Eculizumab

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.
  • Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain.
  • “I’m thrilled to welcome Keith to our Board of Directors, who brings deep industry expertise and invaluable experience to the Rocket Board as we build upon our commercial and operational infrastructure to support our anticipated first product launches for severe Leukocyte Adhesion Deficiency (LAD-I) and Fanconi Anemia (FA),” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Earlier in his career, Mr. Woods held commercial and sales positions of increasing responsibility at Roche, Amgen and Eisai.

A.forall Announces Launch of a Generic Version of Pyridostigmine Bromide Syrup

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Lunedì, Dicembre 11, 2023
FDA, Pharmaceutical, Health, Pyridostigmine, ANDA, Food, Abbreviated New Drug Application, Acella, Patient, Multimedia, Myasthenia gravis, Neurology, Muscle weakness, Muscle fatigue, Generic drug, Pharmaceutical industry

Milla Pharmaceuticals Inc. , an A.forall company, announced that its partner, Acella Pharmaceuticals LLC , has just commercialized its Abbreviated New Drug Application (ANDA) approval from the U.S. Food and Drug Administration (FDA) for a generic version of Mestinon® (Pyridostigmine Bromide Syrup) 60 mg/5mL.

Key Points: 
  • Milla Pharmaceuticals Inc. , an A.forall company, announced that its partner, Acella Pharmaceuticals LLC , has just commercialized its Abbreviated New Drug Application (ANDA) approval from the U.S. Food and Drug Administration (FDA) for a generic version of Mestinon® (Pyridostigmine Bromide Syrup) 60 mg/5mL.
  • View the full release here: https://www.businesswire.com/news/home/20231211602217/en/
    Pyridostigmine Bromide Syrup is indicated for symptomatic treatment of myasthenia gravis.
  • This achievement marks the third ANDA commercialization for Milla Pharmaceuticals Inc. and the fourth for an A.forall product in the U.S. market.
  • With all other projects in our pipeline, our team looks forward to continuing that trend and turning our mission into reality.”
    Acella is excited to partner with Milla Pharmaceuticals on the commercialization of the Pyridostigmine Bromide Syrup.

Food And Drug Administration Grants Two New Label Changes To Tirosint®-Sol (Levothyroxine Sodium) Oral Solution

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Lunedì, Dicembre 4, 2023
Biotechnology, FDA, Managed Care, Health, General Health, Women, Seniors, Pharmaceutical, Research, Consumer, Science, Breakfast, Heat intolerance, Absorption, Food, Hypothyroidism, Eating, Commercial Operating System, Irritability, Headache, Risk, Tremor, Arrhythmia, IBSA, Rash, Cardiovascular disease, Insurance, Patient, Diabetes, Myocardial infarction, Periodic breathing, Appetite, Atrial fibrillation, Muscle weakness, Enzyme inhibitor, Adrenal insufficiency, Diarrhea, Menstruation, University, Water, Insomnia, PPI, Glycerol, Common, Spasm, Nervous, Hyperthyroidism, Weight loss, Pharmacy, Pharmaceutical industry, Dietary supplement, Levothyroxine

The U.S. Food and Drug Administration (FDA) has approved two new changes to the label of Tirosint-SOL (levothyroxine sodium) oral solution, a unique formulation of levothyroxine (LT4) for the treatment of hypothyroidism.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) has approved two new changes to the label of Tirosint-SOL (levothyroxine sodium) oral solution, a unique formulation of levothyroxine (LT4) for the treatment of hypothyroidism.
  • The first regards the use of Tirosint-SOL in the presence of proton pump inhibitor (PPI) therapy.
  • Both label changes help to differentiate Tirosint-SOL from other levothyroxine therapies.
  • Similarly, current labeling for all levothyroxine sodium therapies instructs patients to self-administer the drug once daily, on an empty stomach, one-half to one hour before breakfast.

Charles River and Genetic Cures for Kids Announce Gene Therapy Manufacturing Collaboration

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Martedì, Novembre 28, 2023
Biotechnology, General Health, Medical Devices, Health, Pharmaceutical, Display resolution, Dystonia, NYSE, DNA, GMP, Hope, Entrepreneurship, Tecmar, Degenerative disease, CDMO, Patient, Hereditary spastic paraplegia, Visual impairment, Genetic testing, Muscle weakness, Spasticity, Golden, Seizure, Cerebellar ataxia, Partnership, CRL, Doctor of Philosophy, Parent, HQ, Life, DSM-IV codes, Intellectual disability, Pharmaceutical industry, Vaccine

Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.
  • In recent years, Charles River has significantly broadened its cell and gene therapy portfolio to simplify complex supply chains and meet growing demand for plasmid DNA, viral vector, and cell therapy services.
  • Combined with the Company’s legacy testing capabilities, Charles River offers a comprehensive “concept-to-cure” advanced therapies solution.
  • - Golden Whitrod, Tallulah Moon’s mother and Co-Founder and President, Genetic Cures for Kids, Inc.

Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate Phase 1/2a Study of ARO-DM1 for Type 1 Myotonic Dystrophy

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Martedì, Novembre 28, 2023
Biotechnology, Medical Devices, Health, Pharmaceutical, Clinical Trials, Life expectancy, Safety, Cataract, RNA interference, Exercise, Arrowhead, Clinical trial, DMPK, DM1, Wheelchair, Pharmacokinetics, Muscle weakness, Messenger, Therapy, Ethics committee, MBA, Committee, Wasting, Myotonia, Arrowhead Pharmaceuticals, Patient, Facial muscles, Discovery, Translational medicine, RNA, Pharmaceutical industry

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.

Key Points: 
  • Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.
  • ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene.
  • Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, wheelchairs, and other assistive devices.
  • Pending clearance, Arrowhead intends to proceed with ARODM1-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in up to 48 subjects with DM1.

Teva Announces Approval of a Generic Version of Forteo® (teriparatide injection), in the U.S.

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Venerdì, Novembre 17, 2023
FDA, Pharmaceutical, Health, Medicine, Vomiting, Digoxin, Osteoporosis, Woman, Urine, Antares, NYSE, Bone disease, Calcium, Spine, Prednisone, Constipation, Allergy, History, IQVIA, Patient, Arthralgia, Medication, TEVA, Fracture, Muscle weakness, System, Blood, Tongue, Vitamin, Bone tumor, Nausea, Kidney, Pain, Swelling, Osteosarcoma, Skin, Rat, TASE, Injection, Face, Blood pressure, Generic drug, Dietary supplement, Pharmaceutical industry, Vaccine, Birth control, Injection moulding, Medical imaging, Poultry farming, Phosphorus, Teriparatide, Teva

Teva Pharmaceuticals Inc., a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), announced today the approval of a generic version of Forteo®1, in the United States.

Key Points: 
  • Teva Pharmaceuticals Inc., a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), announced today the approval of a generic version of Forteo®1, in the United States.
  • Currently, 1 in 12 generic prescriptions dispensed in the U.S. is filled with a Teva generic product.
  • Teva’s generic equivalent of Forteo® (teriparatide injection) utilizes the Antares Pharma, Inc. multi-dose pen device.
  • Teriparatide injection can lessen the chance of broken bones (fractures) in the spine and other bones in postmenopausal women with osteoporosis.

Genethon Announces Publication in The Lancet Neurology of Clinical Trial Results of a Gene Therapy for Myotubular Myopathy, a Severe Muscle Disease

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Venerdì, Novembre 17, 2023
Research, Technology, Genetics, Clinical Trials, Nanotechnology, Biotechnology, Pharmaceutical, Health, Science, Other Science, The Lancet, X-linked myotubular myopathy, Acquisition, Therapy, Gene, Pathology, Head, Clinical trial, MTM1, Death, Muscle weakness, Periodic breathing, Muscular Dystrophy Association, Astellas Pharma, Neurology, Myopathy, Knowledge, Lancet Neurology, Pharmaceutical industry, Audentes/Noortekoondis, Inserm

Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.

Key Points: 
  • Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.
  • Characterized by extreme muscle weakness and severe respiratory distress, 50% of affected children die before age 18 months and 75% die before age 10.
  • The gene therapy uses an adeno-associated viral vector (AAV8) to deliver a copy of the MTM1 gene.
  • It took years of research to imagine, design and demonstrate the efficacy of the gene therapy for this very severe and complex disease.

Orphan designation: Dabrafenib mesylate Treatment of glioma, 09/12/2020 Positive

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Lunedì, Dicembre 18, 2023
Glioma, Vomiting, Medicine, Eurostat, COMP, Skin cancer, Pressure, Brain, Safety, Radiation, Merrion Road, European Commission, Ageing, Disease, Headache, Civil service commission, Patient, European, Skull, Dabrafenib, Marketing, Lung cancer, Muscle weakness, Committee, Seizure, Melanoma, BRAF, Population, Nausea, Survival, Cancer, Diagnosis, Date rape drug, Growth, Regulation, Personality, EURORDIS, Knowledge, Tribhuvan University, Cell, Pharmaceutical industry, Medical device, Medical imaging, Vaccine

Orphan designation: Dabrafenib mesylate Treatment of glioma, 09/12/2020 Positive

Key Points: 


Orphan designation: Dabrafenib mesylate Treatment of glioma, 09/12/2020 Positive

HARMONY BIOSCIENCES ANNOUNCES POSITIVE TOPLINE DATA FROM PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT IN ADULT PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1

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Giovedì, Dicembre 7, 2023
Harmony, Safety, ESS, EDS, Narcolepsy, Fatigue, Data, FSS, Patient, DM1, OLE, Cataplexy, FDA, Muscle weakness, DSS, Pitolisant, Epworth Sleepiness Scale, Myotonic dystrophy, Myotonia, News, Somnolence, Pharmaceutical industry, Nursing, Medicinal plants

PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).

Key Points: 
  • PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).
  • "In addition, a positive signal for pitolisant has been demonstrated for fatigue, suggesting it could be a potential new treatment option for this symptom as well.
  • "The positive signals from this Phase 2 study are very encouraging, and support pitolisant as a potential treatment option for DM1."
  • This Phase 2 signal detection study was a randomized, double-blind, placebo-controlled study in adults ages 18-65 with DM1.