Metachromasia

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Retrieved on: 
Monday, March 18, 2024
Encephalitis, Locomotion, Brain, B cell, Bone marrow, Cell, Dietary supplement, Nervous system, Risk, Patient, Public, Urinary tract infection, Animal, Metachromasia, DSM-IV codes, Peripheral nervous system, Viral, Human services, Hope, Death, Quality of life, Atidarsagene autotemcel, Rash, Therapy, Metachromatic leukodystrophy, Natural history, Fever, MLD, Heated tobacco product, Safety, Disability studies, Cosmetics, Health, Vaccine, Food, Swelling, FDA, Liver, Multimedia, ARSA, Pharmaceutical industry

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.

Key Points: 
  • Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.
  • The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene.
  • In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children.
  • Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

Homology Medicines Presents Preclinical Data Supporting Immunosuppression Regimen in Ongoing PKU and Hunter Syndrome Clinical Trials, and Details Optimized MLD Gene Therapy Candidate at the 19th Annual WORLDSymposium™ Meeting

Retrieved on: 
Wednesday, February 22, 2023
Clinical trial, Dexamethasone, Central nervous system, ARSA, Administration, WORLD, MPS II, Brain, Research, PAH, Publication, Phenylketonuria, CNS, Metachromasia, PKU, Gene expression, MPS, JumpStart, Messenger, NAB, MLD, Hunter syndrome, Vaccine

BEDFORD, Mass., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data that supports the targeted, prophylactic immunosuppression regimen in the ongoing pheEDIT gene editing clinical trial in adults with phenylketonuria (PKU) and juMPStart gene therapy trial in adults with Hunter syndrome (MPS II). Homology also shared additional details of the optimized, in vivo gene therapy candidate HMI-204 for metachromatic leukodystrophy (MLD) during the 19th Annual WORLDSymposium™ Meeting.

Key Points: 
  • Homology also shared additional details of the optimized, in vivo gene therapy candidate HMI-204 for metachromatic leukodystrophy (MLD) during the 19th Annual WORLDSymposium™ Meeting.
  • administration in the murine disease model and resulted in levels of enzyme activity predicted to lead to efficacy in vivo.
  • In the poster titled, “Gene Therapy for Metachromatic Leukodystrophy: Lead Candidate Optimization,” a single I.V.
  • Additionally, packaging productivity of HMI-204 demonstrated a substantial improvement in vector genome yields, compared to the earlier MLD gene therapy candidate.

LoQus23 Therapeutics appoints Dr. Melanie Ivarsson, OBE, as Non-Executive Director

Retrieved on: 
Tuesday, January 25, 2022
Entrepreneurship, Research, Service, Metachromasia, Patient, Metachromatic leukodystrophy, Huntington's disease, Disease, Degenerative disease, Neurodegeneration, DNA mismatch repair, Idursulfase, CEO, Hereditary angioedema, COVID-19, Eli Lilly, Gaucher's disease, DNA, Hunter syndrome, Public health, MMR, Therapy, OBE, Company, Lanadelumab, Drug development, Glycogen storage disease, Lists of diseases, Board, Pharmaceutical industry, Pfizer, Moderna, Alfa Romeo, Takeda

CAMBRIDGE, England, Jan. 25, 2022 /PRNewswire/ -- LoQus23 Therapeutics Ltd. ('LoQus23'), a biotech discovering small molecule therapies that target aberrant DNA mismatch repair (MMR) to treat Huntington's and other triplet repeat diseases, announces the appointment of Dr. Melanie Ivarsson, OBE, as Non-Executive Director of the Board.

Key Points: 
  • CAMBRIDGE, England, Jan. 25, 2022 /PRNewswire/ -- LoQus23 Therapeutics Ltd. ('LoQus23'), a biotech discovering small molecule therapies that target aberrant DNA mismatch repair (MMR) to treat Huntington's and other triplet repeat diseases, announces the appointment of Dr. Melanie Ivarsson, OBE, as Non-Executive Director of the Board.
  • David Reynolds, CEO and Co-Founder of LoQus23, said: "On behalf of the board I'd like to welcome Melanie to LoQus23.
  • With these factors combined, LoQus23 has the potential to become a frontrunner in this important disease area."
  • LoQus23 is focused on using a structure-based approach to design small molecule drugs, which can offer more convenient administration than other approaches.

LoQus23 Therapeutics appoints Dr. Melanie Ivarsson, OBE, as Non-Executive Director

Retrieved on: 
Tuesday, January 25, 2022
Entrepreneurship, Research, Service, Metachromasia, Patient, Metachromatic leukodystrophy, Huntington's disease, Disease, Degenerative disease, Neurodegeneration, DNA mismatch repair, Idursulfase, CEO, Hereditary angioedema, COVID-19, Eli Lilly, Gaucher's disease, DNA, Hunter syndrome, Public health, MMR, Therapy, OBE, Company, Lanadelumab, Drug development, Glycogen storage disease, Lists of diseases, Board, Pharmaceutical industry, Pfizer, Moderna, Alfa Romeo, Takeda

CAMBRIDGE, England, Jan. 25, 2022 /PRNewswire/ -- LoQus23 Therapeutics Ltd. ('LoQus23'), a biotech discovering small molecule therapies that target aberrant DNA mismatch repair (MMR) to treat Huntington's and other triplet repeat diseases, announces the appointment of Dr. Melanie Ivarsson, OBE, as Non-Executive Director of the Board.

Key Points: 
  • CAMBRIDGE, England, Jan. 25, 2022 /PRNewswire/ -- LoQus23 Therapeutics Ltd. ('LoQus23'), a biotech discovering small molecule therapies that target aberrant DNA mismatch repair (MMR) to treat Huntington's and other triplet repeat diseases, announces the appointment of Dr. Melanie Ivarsson, OBE, as Non-Executive Director of the Board.
  • David Reynolds, CEO and Co-Founder of LoQus23, said: "On behalf of the board I'd like to welcome Melanie to LoQus23.
  • With these factors combined, LoQus23 has the potential to become a frontrunner in this important disease area."
  • LoQus23 is focused on using a structure-based approach to design small molecule drugs, which can offer more convenient administration than other approaches.

Passage Bio Reports Third Quarter 2021 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Thursday, November 4, 2021
Safety, COVID-19, Central nervous system disease, Research, Charcot–Marie–Tooth disease, University, Metachromasia, Failure, CNS, Data, Amyotrophy, Security (finance), U.S. Securities and Exchange Commission, Patient, Krabbe disease, Investor, Private Securities Litigation Reform Act, Infant, SEC, PASG, Frontotemporal dementia, Risk, Adult, Research and development, Company, Beta-galactosidase, Clinical trial, Webcast, GM1, Temporal lobe epilepsy, IND, Program, R, Nasdaq, Serum, Cerebrospinal fluid, Amyotrophic lateral sclerosis, Metachromatic leukodystrophy, GLOBE, Administration, Pharmaceutical industry

ET

Key Points: 
  • ET
    PHILADELPHIA, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system disorders, today reported financial results for the third quarter ended September 30, 2021 and provided recent business highlights.
  • As we close this year, we remain steadfastly focused on strong execution of our three clinical programs.
  • Appointed Maxine Gowen, Ph.D., as chairwoman of the board of directors in August 2021: This appointment followed the unexpected passing of Passage Bio co-founder and former board chair Dr. Tadataka Yamada.
  • General and Administrative (G&A) Expenses:G&A expenses were $15.0 million for the third quarter ended September 30, 2021, compared to $7.8 million for the same quarter in 2020.

Cure MLD, The Calliope Joy Foundation and NORD Launch A Research and Engagement Study of Metachromatic Leukodystrophy (MLD)

Retrieved on: 
Monday, August 16, 2021
ARSA, Haematopoietic system, Multimedia, Leukodystrophy, Engagement, HIV disease progression rates, PSAP, National Organization for Rare Disorders, Orphan Drug Act of 1983, Prevalence, Awareness, American Community Survey, Caregiver, Degenerative disease, Child, Cal, Community, Adult, MRI, Survey, Metachromasia, Disease, Medicine, Alaska, Brain, Patient, Guardians of the Galaxy, Fats, Parent, Research, Organization, Personality, NORD, Seizure, NIH Intramural Research Program, Metachromatic leukodystrophy, Incidence, Diagnosis, Community Participation, Associate, MLD, Alaska Natives, Urine test strip, Solution, White matter, Enzyme replacement therapy, ASA, Medical device, Pharmaceutical industry, Medical imaging

The new study, Metachromatic Leukodystrophy Community United Research and Engagement Survey (MLDCures), creates a platform for patients around the world to share information about Metachromatic Leukodystrophy (MLD).

Key Points: 
  • The new study, Metachromatic Leukodystrophy Community United Research and Engagement Survey (MLDCures), creates a platform for patients around the world to share information about Metachromatic Leukodystrophy (MLD).
  • "We are thrilled to announce the launch of MLDCures, a patient contact registry for families impacted by metachromatic leukodystrophy," said Maria Kefalas, cofounder of Cure MLD and The Calliope Joy Foundation.
  • To help drive awareness and participation, Cure MLD and The Calliope Joy Foundation will launch an online campaign that demonstrates how the survey will be used and how individuals can register.
  • Cure MLD and The Calliope Joy Foundation are members of NORD and the organizations work together to eliminate the challenges that rare disease patients face.

Metachromatic Leukodystrophy (MLD) Market Assessment, 2020 - Pipeline Products, Unmet Needs, Market Drivers & Barriers, Competitive Landscape

Retrieved on: 
Thursday, March 12, 2020
Neurological disorders, Rare diseases, Organ systems, Lipid storage disorders, Nervous system, Leukodystrophy, Metachromatic leukodystrophy, MLD, Metachromasia

DUBLIN, March 12, 2020 /PRNewswire/ -- The "Metachromatic Leukodystrophy (MLD) - Competitive Landscape, Market and Pipeline Analysis, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, March 12, 2020 /PRNewswire/ -- The "Metachromatic Leukodystrophy (MLD) - Competitive Landscape, Market and Pipeline Analysis, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • Metachromatic Leukodystrophy (MLD) - Competitive Landscape, Market and Pipeline Analysis, 2020 provides comprehensive insights on the therapeutic development for this mechanism of action.
  • The objective of the report is to establish an understanding of the therapeutic competitive landscape for Metachromatic Leukodystrophy (MLD), including the pipeline products in this space.
  • The report provides the understanding of the unmet needs, market drivers and barriers of the Metachromatic Leukodystrophy market.