Neurofibromatosis type I

Ashvattha Therapeutics Announces Presentation of Preclinical Data at the 2022 Neurofibromatosis (NF) Conference Demonstrating the Potential of Hydroxyl Dendrimer Therapeutics to Reduce Toxicity in Targeted Treatment of Plexiform Neurofibroma

Retrieved on: 
Tuesday, June 21, 2022
NF1, Skin, American Academy, Lists of diseases, Therapy, Pediatrics (journal), Microglia, Safety, Neurofibromatosis type I, Tumor-associated macrophage, Neuron, HDT, Technology, HD, Tumor microenvironment, Inherited disorders of trafficking, CEO, Diabetic retinopathy, Toxicity, Johns Hopkins University, Indiana University School of Medicine, HDS, Brain, AMD, Neuro, Inflammation, Ophthalmology, Neurofibroma, Growth, Disease, Neurofibromatosis, TME, Ashvattha, Dendrimer, DME, NF, Patient, Neoplasm, Pigment, Conference, Macrophage, Neurology, GLOBE, PNF, Tams, PSFS Building, Pharmaceutical industry

The data was presented by Emma C. Mazurek, a scientific collaborator at Indiana University School of Medicine, in a poster titled, Hydroxyl Dendrimer Therapeutics Reduce Toxicity in Targeted Delivery to Plexiform Neurofibroma.

Key Points: 
  • The data was presented by Emma C. Mazurek, a scientific collaborator at Indiana University School of Medicine, in a poster titled, Hydroxyl Dendrimer Therapeutics Reduce Toxicity in Targeted Delivery to Plexiform Neurofibroma.
  • Notably, the smaller HD (~14 kDa) showed greater uptake in tumor associated macrophages and microglia, than the larger HD (~56 kDa).
  • These results suggest that by conjugating drugs to HDs theres potential to improve safety and reduce toxicity associated with current treatments for NF.
  • Ashvattha Therapeutics is a clinical-stage biotech company developing novel hydroxyl dendrimer therapeutics (HDTs) targeting unmet medical needs in neurology, ophthalmology, inflammatory diseases and neuro-oncology.

Ashvattha Therapeutics Announces Poster Presentation of Preclinical Data on Hydroxyl Dendrimer-Based Therapeutics at the 2022 Neurofibromatosis (NF) Conference

Retrieved on: 
Tuesday, June 7, 2022
Neurofibromatosis, HDS, PSFS Building, Conference, Toxicity, Johns Hopkins University, Inflammation, NF, Disease, Neurofibromatosis type I, AMD, Therapy, Diabetic retinopathy, NF1, Neuro, HDT, Ophthalmology, GLOBE, Neurology, Technology, COVID-19, DME, Ashvattha, Pharmaceutical industry

REDWOOD CITY, Calif., June 07, 2022 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics (Ashvattha), a clinical stage company developing novel hydroxyl dendrimer therapeutics, today announced a poster presentation of preclinical data demonstrating its hydroxyl dendrimer-based therapeutics reduce toxicity in targeted delivery to plexiform neurofibroma, a slow growing tumor associated with neurofibromatosis type 1 (NF1), at the 2022 Neurofibromatosis (NF) Conference hosted by the Childrens Tumor Foundation and taking place at Loews Philadelphia Hotel in Philadelphia, PA, June 18 21, 2022.

Key Points: 
  • REDWOOD CITY, Calif., June 07, 2022 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics (Ashvattha), a clinical stage company developing novel hydroxyl dendrimer therapeutics, today announced a poster presentation of preclinical data demonstrating its hydroxyl dendrimer-based therapeutics reduce toxicity in targeted delivery to plexiform neurofibroma, a slow growing tumor associated with neurofibromatosis type 1 (NF1), at the 2022 Neurofibromatosis (NF) Conference hosted by the Childrens Tumor Foundation and taking place at Loews Philadelphia Hotel in Philadelphia, PA, June 18 21, 2022.
  • Poster presentation details can be found below:
    Ashvattha Therapeutics is a clinical-stage biotech company developing novel hydroxyl dendrimer therapeutics (HDTs) targeting unmet medical needs in neurology, ophthalmology, inflammatory diseases, and neuro-oncology.
  • The therapies are based on hydroxyl dendrimers (HDs), a targeted platform technology exclusively licensed from our founders, Kannan Rangaramanujam and Sujatha Kannan at Johns Hopkins University.
  • HDs chemically conjugated to disease modifying drugs create novel proprietary HD therapeutics (HDTs) selectively targeting reactive inflammatory cells in disease tissue with localized sustained effects.

SpringWorks Therapeutics Announces Phase 1b/2a Clinical Trial of Mirdametinib in Patients with Advanced Solid Cancers Harboring MAPK-Activating Mutations

Retrieved on: 
Tuesday, August 3, 2021
Branches of biology, Clinical medicine, Organ systems, Neurofibroma, Neurofibromin 1, Antineoplastic drugs, Genodermatoses, MEK inhibitor, Targeted therapy, Neurofibromatosis type I

Hormonal therapies targeting ER, such as SERDs, can be effective in treating ER+ mBC, but over 90% of patients eventually develop resistance to ER-targeted therapy.

Key Points: 
  • Hormonal therapies targeting ER, such as SERDs, can be effective in treating ER+ mBC, but over 90% of patients eventually develop resistance to ER-targeted therapy.
  • We are committed to exploring the full potential of mirdametinib on behalf of patients with devastating cancers and look forward to collaborating with Dr. Rosen and his colleagues at MSK on this important trial.
  • In addition, mirdametinib is being evaluated in a Phase 1b/2 trial in combination with BeiGenes RAF dimer inhibitor, lifirafenib, in patients with advanced or refractory solid tumors harboring RAS mutations, RAF mutations, and other MAPK pathway aberrations.
  • NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas.

Celebrities Support the Children's Tumor Foundation in Raising Awareness and Funds for Rare Genetic Disorder

Retrieved on: 
Friday, May 14, 2021
Organ systems, Genodermatoses, Neurofibromatosis, Branches of biology, Nervous system, Children's Tumor Foundation, Neurofibroma, NF, Neurofibromatosis type I

NF, short for neurofibromatosis, is a rare genetic disorder that causes tumors to grow on nerves throughout the body and affects all ethnicities, races, and genders equally.

Key Points: 
  • NF, short for neurofibromatosis, is a rare genetic disorder that causes tumors to grow on nerves throughout the body and affects all ethnicities, races, and genders equally.
  • In addition to tumors growing anywhere in the body, NF may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer.
  • In some patients, NF is invisible; healthy-looking patients on the outside live with excruciating pain on the inside.
  • There is no cure yet but the Children's Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF.

H1 2020 Pipeline Review on Neurofibromatoses Type I (Von Recklinghausen's Disease) - ResearchAndMarkets.com

Retrieved on: 
Wednesday, October 14, 2020
Health, Genetics, Neurofibromatosis type I, Pipeline, Health, Clinical medicine, Medical specialties, Genodermatoses

The "Neurofibromatoses Type I (Von Recklinghausen's Disease) - Pipeline Review, H1 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Neurofibromatoses Type I (Von Recklinghausen's Disease) - Pipeline Review, H1 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The Neurofibromatoses Type I (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Neurofibromatoses Type I (Von Recklinghausen's Disease) and features dormant and discontinued projects.
  • The pipeline guide evaluates Neurofibromatoses Type I (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • Formulate corrective measures for pipeline projects by understanding Neurofibromatoses Type I (Genetic Disorders) pipeline depth and focus of Indication therapeutics.

Stars Come Together to Help Kids with Rare Genetic Disorder That Causes Tumors

Retrieved on: 
Friday, May 15, 2020
RTT, Genodermatoses, Organ systems, Health, Neuro-cardio-facial-cutaneous syndromes, Neurofibromatosis, Clinical medicine, NF, Disfigurement, Cancer, Neurofibromatosis type I

NF, short for neurofibromatosis, is a rare genetic disorder that causes tumors to grow on nerves throughout the body and affects all ethnicities, races, and genders equally.

Key Points: 
  • NF, short for neurofibromatosis, is a rare genetic disorder that causes tumors to grow on nerves throughout the body and affects all ethnicities, races, and genders equally.
  • In addition to tumors growing anywhere in the body, NF may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer.
  • NF patients will also share their stories about why making NF visible is more important now than ever before.
  • NF causes tumors to grow on nerves throughout the body and may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer.

FDA Approves First Ever Treatment for Neurofibromatosis

Retrieved on: 
Monday, April 13, 2020
Organ systems, Genodermatoses, Nervous system, RTT, Neurofibroma, Children's Tumor Foundation, Neurofibromin 1, NF1, Neuro-cardio-facial-cutaneous syndromes, Neurofibromatosis type I

Affecting 1 in 3,000 people of all populations equally, this announcement is the first ever approved treatment for NF, and portends the potential for the development of treatment options for all NF patients.

Key Points: 
  • Affecting 1 in 3,000 people of all populations equally, this announcement is the first ever approved treatment for NF, and portends the potential for the development of treatment options for all NF patients.
  • In those clinical trials, over 70% of NF patients with inoperable plexiform neurofibromas saw tumor size reduction anywhere from 20-60% in size.
  • "We believe that FDA approval of this treatment helps not only a subset of NF1 patients, it opens the door to increased interest in all forms of NF by pharmaceutical companies.
  • Founded in 1978, the Children's Tumor Foundation (CTF) is the world's largest nongovernmental funder of all forms of neurofibromatosis research in the world.

Children's Tumor Foundation Hosts NF Innovation Week in San Francisco

Retrieved on: 
Friday, September 13, 2019
Children's Tumor Foundation, NF, Neurofibromatosis type I

The Children's Tumor Foundation, the nonprofit global leader in NF research, is harnessing this revitalized interest in rare disease research through a series of events being held in San Francisco, called NF Innovation Week.

Key Points: 
  • The Children's Tumor Foundation, the nonprofit global leader in NF research, is harnessing this revitalized interest in rare disease research through a series of events being held in San Francisco, called NF Innovation Week.
  • This is done through the Foundation's Synodos research teams, the NF Data Portal, NF Registry, and NF Platform, among others.
  • Even more unique, with patients coming into town for the NF Forum, they will also participate at the science events (NF Hackathon and NF Conference).
  • There is no cure yet but the Children's Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF.