Junctional epidermolysis bullosa

Eloxx Pharmaceuticals Reports First Quarter 2021 Financial and Operating Results and Provides Business Update

Retrieved on: 
Thursday, May 6, 2021
Genodermatoses, Rare diseases, Junctional epidermolysis bullosa, Health, Epidermolysis bullosa, Branches of biology, Investigational New Drug, Clinical medicine, Epidermolysis bullosa dystrophica

Our enthusiasm has only increased in the short period since the acquisition,\xe2\x80\x9d said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.

Key Points: 
  • Our enthusiasm has only increased in the short period since the acquisition,\xe2\x80\x9d said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • The acquisition also adds a preclinical stage pipeline in rare diseases and oncology targeting RNA and ribosomal mutations.
  • Eloxx expects to file an Investigational New Drug application for the first oral drug to treat patients with Recessive Dystrophic and Junctional Epidermolysis Bullosa (RDEB and JEB) in 2022.
  • Eloxx\xe2\x80\x99s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins.

Global Epidermolysis Bullosa Epidemiology and Patient Flow Report 2020-2035 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 5, 2021
Health, Other Health, Genodermatoses, Rare diseases, Epidermolysis bullosa, Branches of biology, Epidermolysis bullosa dystrophica, Junctional epidermolysis bullosa

b'The "Global Epidermolysis Bullosa Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Epidermolysis Bullosa Epidemiology and Patient Flow Analysis - 2021, provides Epidermolysis Bullosa epidemiology, demographics, and patient flow.

Key Points: 
  • b'The "Global Epidermolysis Bullosa Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nGlobal Epidermolysis Bullosa Epidemiology and Patient Flow Analysis - 2021, provides Epidermolysis Bullosa epidemiology, demographics, and patient flow.
  • The data is presented by Pharma G7 countries including the US, France, Spain, Italy, UK, Japan.\nThe research provides population data to characterize Epidermolysis Bullosa patients, history of the disease at the population level (Epidermolysis Bullosa prevalence, Epidermolysis Bullosa incidence) and at the clinical level (from diagnosis to treated patients).
  • It also helps to identify patient sub-groups (age, gender, sub-groups) to understand targeted population for research and development, commercialization.\nDemographics: Epidermolysis Bullosa patients by age group, gender\nThe data from this research will help executives:\nEstablish basis for Epidermolysis Bullosa market sizing, assessing market potential, and developing drug forecast models\nIdentify Epidermolysis Bullosa patients segments through age groups, gender, and disease sub-types\nEvaluate Epidermolysis Bullosa market opportunities, identify target patient population\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210505005468/en/\n'

Epidermolysis bullosa Market Size is Anticipated to Accelerate with a CAGR of 5.67% in the 7MM for the Study Period 2017-30

Retrieved on: 
Wednesday, November 18, 2020
Genodermatoses, Rare diseases, Epidermolysis bullosa, Branches of biology, Clinical medicine, Health, Junctional epidermolysis bullosa, Lethal acantholytic epidermolysis bullosa

Research Highlights from the Epidermolysis bullosa Market report:

Key Points: 
  • Research Highlights from the Epidermolysis bullosa Market report:
    The United States accounts for the highest Epidermolysis bullosa market size.
  • According to DelveInsight's Epidermolysis market evaluation, among all the gene therapies in the pipeline, Beremagene geperpavec (KB103) is anticipated to cover a decent market share.
  • Epidermolysis bullosa Market Report proffers 3-year historical as well as 11-year forecasted epidemiological analysis segmented into:
    Total Epidermolysis bullosa Prevalent Cases,
    Diagnosed Epidermolysis bullosa Prevalent Patient Population, and
    The present Epidermolysis bullosa treatment market is limited to palliative care.
  • The increasing attention towards the genetic, rare skin condition is anticipated to yield dramatic results in transforming the Epidermolysis bullosa market landscape.

Epidermolysis bullosa Market Size is Anticipated to Accelerate with a CAGR of 5.67% in the 7MM for the Study Period 2017-30

Retrieved on: 
Wednesday, November 18, 2020
Genodermatoses, Rare diseases, Epidermolysis bullosa, Branches of biology, Clinical medicine, Health, Junctional epidermolysis bullosa, Lethal acantholytic epidermolysis bullosa

Research Highlights from the Epidermolysis bullosa Market report:

Key Points: 
  • Research Highlights from the Epidermolysis bullosa Market report:
    The United States accounts for the highest Epidermolysis bullosa market size.
  • According to DelveInsight's Epidermolysis market evaluation, among all the gene therapies in the pipeline, Beremagene geperpavec (KB103) is anticipated to cover a decent market share.
  • Epidermolysis bullosa Market Report proffers 3-year historical as well as 11-year forecasted epidemiological analysis segmented into:
    Total Epidermolysis bullosa Prevalent Cases,
    Diagnosed Epidermolysis bullosa Prevalent Patient Population, and
    The present Epidermolysis bullosa treatment market is limited to palliative care.
  • The increasing attention towards the genetic, rare skin condition is anticipated to yield dramatic results in transforming the Epidermolysis bullosa market landscape.

Global Epidermolysis Bullosa Pipeline 2020: Drugs, Companies, Clinical Trials, R&D Updates, Status and Outlook - ResearchAndMarkets.com

Retrieved on: 
Tuesday, August 4, 2020
Pharmaceutical, Health, Clinical trials, Genodermatoses, Rare diseases, Epidermolysis bullosa, RTT, Pipeline, Protein replacement therapy, Junctional epidermolysis bullosa

The "Epidermolysis Bullosa Pipeline Research Monitor, 2020 - Drugs, Companies, Clinical Trials, R&D Pipeline Updates, Status and Outlook" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Epidermolysis Bullosa Pipeline Research Monitor, 2020 - Drugs, Companies, Clinical Trials, R&D Pipeline Updates, Status and Outlook" report has been added to ResearchAndMarkets.com's offering.
  • Global Epidermolysis Bullosa market presents promising new product pipeline with NME Projects, pivotal trials, and rapidly phase-advancing therapeutic candidates.
  • This Epidermolysis Bullosa pipeline review explores high-potential early to late-stage pipeline projects with a continued focus on new insights, accelerated processes, and pipeline progression.
  • The competitive intelligence report on Epidermolysis Bullosa presents detailed insights into therapeutic drug pipeline development, industry news, deals, and analysis across the length and breadth of the Epidermolysis Bullosa pipeline.

BridgeBio Pharma’s Phoenix Tissue Repair Provides Updates to its Recessive Dystrophic Epidermolysis Bullosa (RDEB) Program and Announces New Leadership Appointments

Retrieved on: 
Wednesday, May 13, 2020
Genodermatoses, Rare diseases, Branches of biology, Collagen disease, Epidermolysis bullosa dystrophica, Structural proteins, Cell biology, Epidermolysis bullosa, Collagen, Junctional epidermolysis bullosa

Intravenous collagen 7 replacement therapy with PTR-01 is the only treatment in development designed to provide broad disease-modifying effects for this multisystem disease.

Key Points: 
  • Intravenous collagen 7 replacement therapy with PTR-01 is the only treatment in development designed to provide broad disease-modifying effects for this multisystem disease.
  • Phoenix Tissue Repair is aBoston-based company that is an affiliate of BridgeBio Pharma, and is focused on advancing a novel systemic treatment for recessive dystrophic epidermolysis bullosa (RDEB).
  • Moreover, Phoenix Tissue Repair operates in a very competitive and rapidly changing environment in which new risks emerge from time to time.
  • All forward-looking statements in this press release are based on information available to Phoenix Tissue Repair as of the date hereof, and Phoenix Tissue Repair disclaims any obligation to update these forward-looking statements except as required by law.

PolarityTE to Sponsor Epidermolysis Bullosa Research Partnership Event "ACTion for Jackson" in New York

Retrieved on: 
Wednesday, November 7, 2018
Genodermatoses, Rare diseases, Epidermolysis bullosa, RTT, EB, Genetic genealogy, Medical genetics, Epidermolysis bullosa dystrophica, Junctional epidermolysis bullosa

During the event, Nikolai Sopko, MD, PhD, Chief Scientific Officer of PolarityTE will discuss the research PolarityTE is conducting in epidermolysis bullosa, a life-threatening rare genetic disease.

Key Points: 
  • During the event, Nikolai Sopko, MD, PhD, Chief Scientific Officer of PolarityTE will discuss the research PolarityTE is conducting in epidermolysis bullosa, a life-threatening rare genetic disease.
  • Epidermolysis bullosa is one of many debilitating genetic diseases that can affect the skin as well as other organs.
  • We hope that one day we will be able to provide a permanent treatment for epidermolysis bullosa and give these patients back their skin."
  • POLARITYTE, the POLARITYTE logo, WHERE SELF REGENERATES SELF, WELCOME TO THE SHIFT, and SKINTE are trademarks or registered trademarks of PolarityTE, Inc.