Peroxisomal disorders

SwanBio Therapeutics Announces First Patient Enrolled in Natural History Study to Evaluate Patients with Adrenomyeloneuropathy

Retrieved on: 
Tuesday, June 29, 2021
Mental health, Health, Genetics, Physical therapy, Other Health, General Health, Clinical trials, Neurological disorders, Health, Rare diseases, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, ALD, Clinical trial, AMN, Adrenomyeloneuropathy, SwanBio Therapeutics, ADRENOMYELONEUROPATHY, SWANBIO THERAPEUTICS

CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.

Key Points: 
  • CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.
  • This observational, multinational study will prospectively evaluate patients to assess the course of the disease and provide insights into potential endpoints and designs for future clinical trials.
  • We are pleased to initiate this natural history study for AMN, which has been strategically designed with patients in mind, and allows for the majority of monitoring to be conducted virtually from each patients home.
  • The global study will enroll approximately 80 adult male patients with a confirmed diagnosis of adrenoleukodystrophy (ALD).

bluebird bio Presents Long-Term Data for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)

Retrieved on: 
Monday, March 15, 2021
Oncology, Health, FDA, Genetics, Clinical trials, Biotechnology, Branches of biology, Neurological disorders, Biology, Rare diseases, Peroxisomal disorders, Bluebird bio, Adrenoleukodystrophy, ATP-binding cassette transporters, Gene therapy, Leukodystrophy, ABCD1, Very long chain fatty acid

The addition of the functional ABCD1 gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to activate the breakdown of VLCFAs.

Key Points: 
  • The addition of the functional ABCD1 gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to activate the breakdown of VLCFAs.
  • All reported data below from ALD-102 are as of October 2020 and all reported data below from LTF-304 are as of November 2020.
  • In October 2020, the European Medicines Agency (EMA) accepted bluebird bios marketing authorization application (MAA) for its investigational eli-cel gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD).
  • eli-cel, Lenti-D and bluebird bio are trademarks of bluebird bio, Inc.

Adrenoleukodystrophy Pipeline Review, H2 2020 Edition - Therapeutics Development and Assessment, Drug Profiles, Dormant Projects, Product Development Milestones, Competitive Landscape - ResearchAndMarkets.com

Retrieved on: 
Tuesday, January 12, 2021
Genetics, Pharmaceutical, Health, Neurological disorders, Rare diseases, Organ systems, Branches of biology, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy

Adrenoleukodystrophy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.

Key Points: 
  • Adrenoleukodystrophy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • The Adrenoleukodystrophy (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) and features dormant and discontinued projects.
  • The pipeline guide reviews pipeline therapeutics for Adrenoleukodystrophy (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • Formulate corrective measures for pipeline projects by understanding Adrenoleukodystrophy (Genetic Disorders) pipeline depth and focus of Indication therapeutics.

Global Adrenoleukodystrophy (ALD) Epidemiology Forecast to 2030 - ResearchAndMarkets.com

Retrieved on: 
Friday, December 11, 2020
Retail, Other Retail, Neurological disorders, Rare diseases, Organ systems, Branches of biology, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, ALD, Epidemiology

The "Adrenoleukodystrophy (ALD) - Epidemiology Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Adrenoleukodystrophy (ALD) - Epidemiology Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.
  • This 'Adrenoleukodystrophy (ALD) - Epidemiology Forecast to 2030' report delivers an in-depth understanding of the disease, historical and forecasted Adrenoleukodystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
  • As per the publisher's analysis, in 2017, the total prevalent population of Adrenoleukodystrophy in the 7MM was 55,242.
  • What will be the growth opportunities in the 7MM concerning the patient population of Adrenoleukodystrophy?

bluebird bio Presents New Results from Clinical Development Program of elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD), Including Updated Long-Term Data, at the 46th Annual Meeting of the EBMT

Retrieved on: 
Saturday, August 29, 2020
FDA, Other Health, Stem cells, Oncology, Managed care, Mental health, Infectious diseases, Genetics, Clinical trials, Cardiology, Science, Biotechnology, Other Science, Health, Research, Rare diseases, Neurological disorders, Organ systems, Clinical medicine, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, ALD

Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide.

Key Points: 
  • Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide.
  • Approximately 40% of boys with adrenoleukodystrophy will develop CALD, the most severe form of ALD.
  • Nearly half of boys with CALD who do not receive treatment will die within five years of symptom onset.
  • These results presented at EBMT 2020 are very encouraging and suggest treatment with eli-cel may prevent neurological decline in boys with CALD.

Adrenoleukodystrophy Pipeline Insights, 2020 - ResearchAndMarkets.com

Retrieved on: 
Monday, April 20, 2020
Health, Pharmaceutical, Neurological disorders, Organ systems, Rare diseases, Health, Peroxisomal disorders, Leukodystrophies, Adrenoleukodystrophy, Drug pipeline

The "Adrenoleukodystrophy - Pipeline Insight - 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Adrenoleukodystrophy - Pipeline Insight - 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • Adrenoleukodystrophy Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Adrenoleukodystrophy market.
  • A detailed picture of the Adrenoleukodystrophy pipeline landscape is provided, which includes the disease overview and Adrenoleukodystrophy treatment guidelines.
  • The assessment part of the report embraces in-depth Adrenoleukodystrophy commercial assessment and clinical assessment of the Adrenoleukodystrophy pipeline products from the pre-clinical developmental phase to the marketed phase.

Global Adrenoleukodystrophy (ALD) Market Study 2020: Drivers, Barriers, Drugs, Unmet Needs and Opportunities

Retrieved on: 
Tuesday, March 24, 2020
Rare diseases, Neurological disorders, Organ systems, Nervous system, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, Market, ALD

The Adrenoleukodystrophy (ALD) market report provides current treatment practices, emerging drugs, and Adrenoleukodystrophy market share of the individual therapies, current and forecasted Adrenoleukodystrophy market size from 2017 to 2030 segmented by seven major markets.

Key Points: 
  • The Adrenoleukodystrophy (ALD) market report provides current treatment practices, emerging drugs, and Adrenoleukodystrophy market share of the individual therapies, current and forecasted Adrenoleukodystrophy market size from 2017 to 2030 segmented by seven major markets.
  • The report also covers current Adrenoleukodystrophy treatment practice, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
  • This section provides the total Adrenoleukodystrophy market size and market size by therapies in the United States.
  • The total Adrenoleukodystrophy market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

Retrieved on: 
Wednesday, September 18, 2019
Stem cells, Biotechnology, General Health, Health, Genetics, Clinical trials, Branches of biology, Rare diseases, Neurological disorders, Organ systems, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, Gene therapy

bluebird bio, Inc. (Nasdaq: BLUE) today announced updated results from the clinical development program for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress in Athens, Greece.

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) today announced updated results from the clinical development program for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress in Athens, Greece.
  • The Phase 2/3 Starbeam study (ALD-102) is assessing the efficacy and safety of Lenti-D in boys 17 years of age and under with CALD.
  • The primary efficacy endpoint in the study is the proportion of patients who are alive and free of MFDs at Month 24.
  • The 14 patients currently on study have less than 24 months of follow-up and have shown no evidence of MFDs.

bluebird bio to Present Data from Clinical Development Program of Lenti-DTM Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

Retrieved on: 
Tuesday, September 3, 2019
Stem cells, Biotechnology, Pharmaceutical, Oncology, Health, Genetics, Branches of biology, Biology, Genetics, Rare diseases, Gene delivery, Peroxisomal disorders, Fatty acids, Very long chain fatty acid, Adrenoleukodystrophy, Leukodystrophy, Gene therapy, ABCD1

bluebird bio, Inc. (Nasdaq: BLUE) announced that updated data from the clinical development program for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD) will be presented at the 13th European Pediatric Neurology Society (EPNS) Congress, to be held September 17 21, 2019 in Athens, Greece.

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) announced that updated data from the clinical development program for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD) will be presented at the 13th European Pediatric Neurology Society (EPNS) Congress, to be held September 17 21, 2019 in Athens, Greece.
  • Gene therapy for CALD consists of the patients own stem cells transduced with Lenti-D lentiviral vector that encodes ABCD1 cDNA for human adrenoleukodystrophy protein (ALDP).
  • This allows for the production of functional ALDP that enables the local degradation of very long chain fatty acids (VLCFAs) in the brain.
  • Buildup of VLCFAs in the central nervous system contributes to neurodegeneration in CALD.