Neuromuscular Disorders

Lupin and Exeltis Announce Reimbursement Approval of NaMuscla® in Spain for the Symptomatic Treatment of Myotonia in Adults with non-dystrophic myotonic (NDM) Disorders

Retrieved on: 
Monday, January 9, 2023
Disease, News, Myotonia, NDM, Lupinus, Degenerative disease, National Health, Quality of life, Safety, Neuromuscular Disorders, Patient, Prevalence, Mexiletine, Investment, Health insurance, Pharmaceutical industry, Renewable energy, EU

NaMuscla® is the first and only licensed product for this indication in Europe and will be commercialized by Lupin’s partner Exeltis in Spain.

Key Points: 
  • NaMuscla® is the first and only licensed product for this indication in Europe and will be commercialized by Lupin’s partner Exeltis in Spain.
  • NDM disorders are a group of rare, inherited neuromuscular disorders with an estimated prevalence of 1 case per 100,000 inhabitants in Spain1, and is characterized by the inability to relax muscles following voluntary contraction (myotonia).
  • NaMuscla® reduces myotonia symptoms in people with NDM, resulting in a significant improvement in quality of life and other functional and clinical outcomes for patients2.
  • We are grateful to all stakeholders involved in the approval process, especially our partner Exeltis,” said Thierry Volle, President EMEA, Lupin.

Revance Provides an Update on DAXXIFY® Commercial Launch and Preliminary Fourth Quarter and Full Year 2022 Financial Results

Retrieved on: 
Monday, January 9, 2023
Biotechnology, FDA, Pharmaceutical, Health, Speech, Spasticity, Education, Therapy, GAAP, Death, Neuromuscular Disorders, Faculty, Patient, Growth, Food, Hubli railway division, Traction, Investment, Neurotoxin, Dysphagia, Pharmaceutical industry, Video game

Revance Therapeutics, Inc. (RVNC) today provided an update on its early experience program for DAXXIFY® and its preliminary unaudited fourth quarter and full year 2022 financial results.

Key Points: 
  • Revance Therapeutics, Inc. (RVNC) today provided an update on its early experience program for DAXXIFY® and its preliminary unaudited fourth quarter and full year 2022 financial results.
  • Revance expects preliminary unaudited DAXXIFY® revenue from PrevU, its early experience program, to be between $10.5 million and $11.5 million in the fourth quarter 2022, the first quarter of limited commercial availability.
  • Revance expects preliminary unaudited fourth quarter 2022 service revenue from OPUL® and the legacy HintMD fintech platform to be between $2.5 million and $3.5 million and full year 2022 preliminary unaudited service revenue of between $6.5 million and $7.5 million.
  • The company expects to provide its 2023 GAAP and Non-GAAP operating expense guidance in its fourth quarter 2022 earnings announcement.

Mike Heffernan appointed Chairman of NMD Pharma’s Board of Directors

Retrieved on: 
Tuesday, January 3, 2023
Eli Lilly and Company, Bausch Health, CM, AbbVie, University, Eli Lilly, Neuromuscular Disorders, Patient, NYSE, NMD, Fine chemical, Pharmaceutical industry, Pharmacy, Collegium (ancient Rome)

Aarhus, Denmark, 3 January 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that Mike Heffernan has been appointed as Chairman of the Board.

Key Points: 
  • Aarhus, Denmark, 3 January 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that Mike Heffernan has been appointed as Chairman of the Board.
  • Mr. Heffernan succeeds Patrick Vink, who has served as NMD Pharma’s Chairman since 2015.
  • He is the CEO and Chairman of the US-based oncology company Avenge Bio, Inc. which he co-founded in 2019.
  • Mike Heffernan, NMD Pharma’s new Chairman commented: “I feel very privileged to take on this role at such an exciting time for NMD Pharma.

Mike Heffernan appointed Chairman of NMD Pharma’s Board of Directors

Retrieved on: 
Tuesday, January 3, 2023
Eli Lilly and Company, Bausch Health, CM, AbbVie, University, Eli Lilly, Neuromuscular Disorders, Patient, NYSE, NMD, Fine chemical, Pharmaceutical industry, Pharmacy, Collegium (ancient Rome)

Aarhus, Denmark, 3 January 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that Mike Heffernan has been appointed as Chairman of the Board.

Key Points: 
  • Aarhus, Denmark, 3 January 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that Mike Heffernan has been appointed as Chairman of the Board.
  • Mr. Heffernan succeeds Patrick Vink, who has served as NMD Pharma’s Chairman since 2015.
  • He is the CEO and Chairman of the US-based oncology company Avenge Bio, Inc. which he co-founded in 2019.
  • Mike Heffernan, NMD Pharma’s new Chairman commented: “I feel very privileged to take on this role at such an exciting time for NMD Pharma.

Revance Announces U.S. FDA Acceptance of Supplemental Biologics License Application (sBLA) for DAXXIFY® (DaxibotulinumtoxinA-lanm) for Injection for the Treatment of Cervical Dystonia

Retrieved on: 
Friday, January 6, 2023
Biotechnology, FDA, Pharmaceutical, Health, Prescription Drug User Fee Act, PDUFA, Food, Muscle, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Death, ASPEN, Injection, Torticollis, Patient, Neuromuscular Disorders, Speech, Physician, Safety, Neurotoxin, FDA, Dysphagia, Spasticity, Hubli railway division, Pharmaceutical industry, MD

Results from the ASPEN-OLS study reinforced the safety findings reported from ASPEN-1 study, as well as the efficacy of DAXXIFY® with up to four repeat treatments.

Key Points: 
  • Results from the ASPEN-OLS study reinforced the safety findings reported from ASPEN-1 study, as well as the efficacy of DAXXIFY® with up to four repeat treatments.
  • “Painful symptom re-emergence is very common for patients with cervical dystonia and up until now physicians have not been able to fully address this issue with existing treatment options,” said Peter McAllister, MD.
  • The effects of DAXXIFY® and all botulinum toxin products may spread from the area of injection to produce symptoms consistent with botulinum toxin effects.
  • The effect of administering different botulinum neurotoxins during course of treatment with DAXXIFY® is unknown.

NMD Pharma Receives IND Clearance to Start a Phase II Clinical Trial of NMD670 for the Treatment of Symptoms of Spinal Muscular Atrophy Type 3

Retrieved on: 
Wednesday, December 14, 2022
Spinal muscular atrophy, SMN1, Mortality, Myasthenia gravis, Incidence, Novo Holdings A/S, Food, FDA, Fatigue, ODD, NMD, Aarhus University, Weakness, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Neuromuscular Disorders, SMA, Research, Disease, MG, Patient, Pharmaceutical industry

NMD Pharma A/S, is a clinical-stage biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders.

Key Points: 
  • NMD Pharma A/S, is a clinical-stage biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders.
  • Positive top line data reported from a Phase I/II clinical trial of lead program NMD670 in myasthenia gravis has provided clinical validation of ClC-1 inhibition to restore neuromuscular function.
  • NMD Pharma has raised ~€80 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital.
  • Type 1 (0) is the most severe form of the disease with symptoms appearing at birth or in infancy (before birth, type 0).

NMD Pharma Receives IND Clearance to Start a Phase II Clinical Trial of NMD670 for the Treatment of Symptoms of Spinal Muscular Atrophy Type 3

Retrieved on: 
Wednesday, December 14, 2022
Spinal muscular atrophy, SMN1, Mortality, Myasthenia gravis, Incidence, Novo Holdings A/S, Food, FDA, Fatigue, ODD, NMD, Aarhus University, Weakness, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Neuromuscular Disorders, SMA, Research, Disease, MG, Patient, Pharmaceutical industry

NMD Pharma A/S, is a clinical-stage biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders.

Key Points: 
  • NMD Pharma A/S, is a clinical-stage biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders.
  • Positive top line data reported from a Phase I/II clinical trial of lead program NMD670 in myasthenia gravis has provided clinical validation of ClC-1 inhibition to restore neuromuscular function.
  • NMD Pharma has raised ~€80 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital.
  • Type 1 (0) is the most severe form of the disease with symptoms appearing at birth or in infancy (before birth, type 0).

AIM ImmunoTech Appoints Christopher McAleer, Ph.D. as Scientific Officer

Retrieved on: 
Monday, December 12, 2022
Science, Degenerative disease, MPS, MD, Toxicity, Science Translational Medicine, West Chester University, Disulfiram-like drug, Multi, Research, COVID-19, Biomedical sciences, Central Florida, EAP, SBIR, Rintatolimod, Erasmus MC, Metabolic syndrome, Blog, Science education, Inspectorate, Manuscript, Senior, Publication, Therapy, Molecular biology, Chronic fatigue syndrome, CFS, Special access program, University of Central Florida, Heart, AIM, Neuromuscular Disorders, IPSC, NIH, ISCB Senior Scientist Award, University, Immune system, Disease, Tissue, Patient, Sarcopenia, Chief scientific officer, NYSE, Pancreatic cancer, Microbiology, Physician, ME/CFS, PSLRA, Cancer, Vaccine, Management, Pharmaceutical industry, Chemistry, Hesperus, Biology

OCALA, Fla., Dec. 12, 2022 (GLOBE NEWSWIRE) -- AIM ImmunoTech Inc. (NYSE American: AIM) (“AIM” or the “Company”), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by the SARS-CoV-2 virus, today announced the promotion of Christopher McAleer, Ph.D., from Deputy Scientific Officer to Scientific Officer.

Key Points: 
  • OCALA, Fla., Dec. 12, 2022 (GLOBE NEWSWIRE) -- AIM ImmunoTech Inc. (NYSE American: AIM) (“AIM” or the “Company”), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by the SARS-CoV-2 virus, today announced the promotion of Christopher McAleer, Ph.D., from Deputy Scientific Officer to Scientific Officer.
  • Chris will make an excellent Scientific Officer,” said David Strayer, MD, AIM’s Chief Scientific and Medical Officer.
  • We are pleased to deepen his role with his appointment as Scientific Officer as we continue to advance our pipeline forward.
  • Additionally, Dr. McAleer has authored published manuscripts in several peer-reviewed science and nature journals, book chapters and scientific conference publications.

Emerging Pipeline Therapeutics Across 6 Rare Neuromuscular Diseases: Strategic Partnering and New Product Development will Enable Better Biologics for Personalized and Targeted Treatment - ResearchAndMarkets.com

Retrieved on: 
Tuesday, December 13, 2022
Biotechnology, Pharmaceutical, Health, Myasthenia gravis, Sarepta, Therapy, Spinal muscular atrophy, CIDP, DN, US FDA, DMD, Disorder, Myopathy, SMA, Duchenne muscular dystrophy, NMD, Golodirsen, Motor neuron disease, Patient, Amyotrophic lateral sclerosis, US, Degenerative disease, Research, Neuromuscular Disorders, NMDS, ALS, Diabetic neuropathy, Eteplirsen, Muscle, Cramp, Quality of life, Muscle weakness, RNA, Patent, Pharmaceutical industry, Vaccine

Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class.

Key Points: 
  • Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class.
  • This research, "Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis" explores emerging biologics across these six NMDs.
  • Key biologics emerging for these rare NMDs include stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments.
  • Therefore, biologics are suitably poised to transform the rare NMD landscape with personalized and targeted treatment strategies and will provide life-saving treatment options for fatal neuromuscular conditions.

Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis

Retrieved on: 
Thursday, December 1, 2022
DN, Myasthenia gravis, MG, Cramp, Muscle weakness, RNA, Disorder, Duchenne muscular dystrophy, Therapy, Golodirsen, Patient, Motor neuron disease, Eteplirsen, Neuromuscular Disorders, US, Patent, Muscle, Amyotrophic lateral sclerosis, NMD, Spinal muscular atrophy, DMD, ALS, Diabetic neuropathy, NMDS, SMA, Quality of life, Myopathy, CIDP, US FDA, Degenerative disease, Dietary supplement, Pharmaceutical industry, Health insurance

Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs).

Key Points: 
  • Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs).
  • The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies.
  • Frost & Sullivans research, Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis explores emerging biologics across these six NMDs.Key biologics emerging for these rare NMDs include stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments.
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