Cystic fibrosis

Proteostasis Therapeutics Announces Positive Preliminary Results from Proprietary Doublet in Cystic Fibrosis Patients

Retrieved on: 
Wednesday, October 17, 2018
Biology, Channelopathies, Cystic fibrosis, RTT, Proteostasis, Pakistan Tehreek-e-Insaf, Gastroenterology, Genetic genealogy

PK results were as expected and confirmed a once-a-day dosing profile for both PTI-801 and PTI-808.

Key Points: 
  • PK results were as expected and confirmed a once-a-day dosing profile for both PTI-801 and PTI-808.
  • "Doublet combinations, the current standard of care for many CF patients, are the benchmark for current treatment in cystic fibrosis.
  • These results begin to confirm the exciting clinical effect of PTI's drug pipelinewith more clinical data from both higher doses and proprietary triplet combinations to come."
  • Proteostasis Therapeutics, Inc.is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing.

Proteostasis Therapeutics to Present at the Cantor Global Healthcare Conference

Retrieved on: 
Thursday, September 27, 2018
Biology, Biotechnology, Nature, Protein folding, Homeostasis, Proteomics, Proteostasis, Biopharmaceutical, Cystic fibrosis, Forward-looking statement

A live audio webcast of theCantor Global Healthcare Conference presentation will be available on the Investor Events page in the Investors & Media section of the Company's website, www.proteostasis.com .

Key Points: 
  • A live audio webcast of theCantor Global Healthcare Conference presentation will be available on the Investor Events page in the Investors & Media section of the Company's website, www.proteostasis.com .
  • A replay of the webcast will be available on the Company's website following the presentation.
  • Proteostasis Therapeutics, Inc.is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing.
  • We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Raising Awareness for Childhood Illnesses Through Philanthropic Partnership

Retrieved on: 
Thursday, September 20, 2018
Sammie, Organ transplantation, Cystic fibrosis

The book tells stories through the eyes of children facing autoimmune diseases, cystic fibrosis, heart transplants, and pediatric cancer.

Key Points: 
  • The book tells stories through the eyes of children facing autoimmune diseases, cystic fibrosis, heart transplants, and pediatric cancer.
  • Samantha (Sammie) Hasen is the author of Long Live the Little Ones in collaboration with Ella Koscik, CEO and Owner at MDI Group, who produced the book.
  • The exchange left Sammie with a passion to raise public awareness for children suffering similar experiences.
  • "I'm grateful to have had the opportunity to support Sammie in this endeavor to spread awareness of critical illnesses that children simply shouldn't have to face.

DarioHealth Chosen by Attain Health for Pilot Study to Improve Cystic Fibrosis Related Diabetes Management

Retrieved on: 
Wednesday, August 22, 2018
Pancreas disorders, Cystic fibrosis, Gastroenterology, Channelopathies, RTT, Anatomy, Health, Cystic fibrosis-related diabetes, Cystic Fibrosis Foundation

Attain Health has committed to the pilot by paying for access to the DarioEngage Dashboard to help monitor the DarioHealth sponsored patients.

Key Points: 
  • Attain Health has committed to the pilot by paying for access to the DarioEngage Dashboard to help monitor the DarioHealth sponsored patients.
  • Attain Health and DarioHealth plan to jointly present preliminary findings from the pilot study in at the North American Cystic Fibrosis Conference on October 18-20, 2018 in Denver, Colorado.
  • "Effective diabetes management in cystic fibrosis patients is critical, as there is a 6-fold increase in mortality amongst cystic fibrosis patients who have diabetes, as compared to those who don't.
  • Attain Health is an integrative health coaching and physical performance program focused on patient-centered care, engaging individuals living with cystic fibrosis to optimize their health.

Novel Drugs and Smart Devices for Respiratory Disorders, 2018-2030 - Evaluation of Approx. 135 Novel Drug Candidates Across Various Stages of Development

Retrieved on: 
Friday, August 10, 2018
Medicine, Pulmonology, Health, Chronic lower respiratory diseases, Respiratory therapy, Respiratory diseases, RTT, Channelopathies, Cystic fibrosis, Asthma, Chronic condition, Chronic obstructive pulmonary disease

The Novel Drugs and Smart Devices for Respiratory Disorders, 2018-2030' report provides a comprehensive study on the current landscape of novel therapies and smart devices being evaluated for the treatment of asthma, COPD and cystic fibrosis.

Key Points: 
  • The Novel Drugs and Smart Devices for Respiratory Disorders, 2018-2030' report provides a comprehensive study on the current landscape of novel therapies and smart devices being evaluated for the treatment of asthma, COPD and cystic fibrosis.
  • With several novel drug candidates in development, we anticipate the market to witness continued growth over the next 5-10 years.
  • According to the Forum of International Respiratory Societies, around 4 million individuals die each year due to chronic respiratory diseases.
  • Around 135 novel drug candidates are being evaluated across various stages of development for the treatment of patients suffering from asthma, COPD and cystic fibrosis.

Embleema Launches the First Health Records Blockchain To Give Patients Complete Control Over Their Health Data and Be at the Center of Clinical Research

Retrieved on: 
Wednesday, July 18, 2018
Health, Health informatics, Terms of service, Clinical trial, Design of experiments, Drug discovery, Product testing, Medical privacy, Cystic fibrosis, Medical record, Blockchain, Privacy

All the while allowing patients to maintain sovereignty and control over their most personal data. "

Key Points: 
  • All the while allowing patients to maintain sovereignty and control over their most personal data. "
  • "A major concern of patients with Cystic Fibrosis is that their health data could be sold without their knowledge or consent.
  • Clinical trial participants don't know where their data is going and it contains a huge amount of personal information beyond just their health records.
  • Blockchain allows patients to share their data to aid in research - but on their terms and with full respect to their privacy.

Crossing For A Cure Endurance Paddle Raises $280,000 for Cystic Fibrosis Community

Retrieved on: 
Thursday, June 28, 2018
Medicine, Cystic fibrosis, Channelopathies, RTT, Gastroenterology, Health, Fibrosis

One hundred paddlers participated in the 75-mile endurance paddle from Bimini, Bahamas to South Florida's Lake Worth Pier an increase of 66 paddlers over 2017.

Key Points: 
  • One hundred paddlers participated in the 75-mile endurance paddle from Bimini, Bahamas to South Florida's Lake Worth Pier an increase of 66 paddlers over 2017.
  • Diagnosed with cystic fibrosis at the age of 4, Suit found hope and inspiration in learning of the healing benefits of saltwater for cystic fibrosis patients.
  • This endurance paddle, which also served as a demonstration to Piper of how one can overcome life challenges and face fear, has now evolved into a movement that encompasses the entire cystic fibrosis community.
  • About Crossing For A Cure:
    Crossing For A Cure is the flagship event for Florida-based cystic fibrosis non-profit organization, Piper's Angels.

Novel Drugs and Smart Devices for Respiratory Disorders, 2018 - 2030

Retrieved on: 
Tuesday, June 26, 2018
Medicine, Respiratory therapy, Health, Chronic lower respiratory diseases, Pulmonology, Dosage forms, Chronic obstructive pulmonary disease, Inhaler, Asthma, Cystic fibrosis, Cystic Fibrosis Foundation

Chapter 6 includes detailed profiles of the players that have received approval for their novel therapies targeting asthma, COPD or cystic fibrosis.

Key Points: 
  • Chapter 6 includes detailed profiles of the players that have received approval for their novel therapies targeting asthma, COPD or cystic fibrosis.
  • Chapter 8 features an overview of the market landscape of the key players involved in the development of smart inhalers.
  • Chapter 9 presents detailed analysis of the key trends observed in the development of novel therapies for asthma, COPD and cystic fibrosis.
  • With several approved drugs and late stage clinical (phase II and above) candidates, the market for novel therapeutics is anticipated to grow at a steady pace between 2018 and 2030.

2018 Crossing For A Cure Endurance Paddle to Benefit Cystic Fibrosis Community

Retrieved on: 
Thursday, June 7, 2018
Channelopathies, Cystic fibrosis, RTT, Gastroenterology, Health, Genetic genealogy, Cystic Fibrosis Canada

WEST PALM BEACH, Fla., June 7, 2018 /PRNewswire-PRWeb/ -- The Piper's Angels Foundation has announced its 2018 Crossing For A Cure charity event, a long distance endurance paddle challenge that raises funds and awareness for cystic fibrosis.

Key Points: 
  • WEST PALM BEACH, Fla., June 7, 2018 /PRNewswire-PRWeb/ -- The Piper's Angels Foundation has announced its 2018 Crossing For A Cure charity event, a long distance endurance paddle challenge that raises funds and awareness for cystic fibrosis.
  • As of June 4, 100 people have registered to paddle in the Crossing For A Cure, and have raised more than $197,000.
  • Diagnosed with cystic fibrosis at the age of 4, Suit found hope and inspiration in learning of the healing benefits of saltwater for cystic fibrosis patients.
  • About Crossing For A Cure:
    Crossing For A Cure is the flagship event for Florida-based cystic fibrosis non-profit organization, Piper's Angels.

Proteostasis Announces Positive Data from Ongoing Phase 1 Study of PTI-801 in Cystic Fibrosis Patients on Background Orkambi® Therapy

Retrieved on: 
Wednesday, June 6, 2018
Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, Genetics, Anatomy, Medicine

The randomized, double-blind, placebo-controlled Phase 1 study is being conducted at 30 sites in the U.S. and Europe.

Key Points: 
  • The randomized, double-blind, placebo-controlled Phase 1 study is being conducted at 30 sites in the U.S. and Europe.
  • At the request of participating study sites, up to an additional 10 subjects will be enrolled in the highest dose cohort.
  • The study was designed to evaluate the safety, tolerability and pharmacokinetics (PK) of PTI-801, a third generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
  • We anticipate reporting initial data from our ongoing doublet study of PTI-801 and PTI-808, followed by our triplet study results, all later this year."