Scleroderma

COLOR CHANGES IN EXTREMITIES WHEN COLD USUALLY NOT NORMAL, SAY MEDICAL EXPERTS

Retrieved on: 
Tuesday, September 27, 2022

REDDING, Calif., Sept. 27, 2022 /PRNewswire/ -- As the colder weather sets in, 5-10 percent of Americans will experience episodes of color changes in their fingers or toes, accompanied by spasms of throbbing pain. That response is not normal, according to the Raynaud's Association, which notes that October is Raynaud's Awareness Month.

Key Points: 
  • That response is not normal, according to the Raynaud's Association, which notes that October is Raynaud's Awareness Month.
  • Typically, the affected area turns white, blue and red over the course of the attack.
  • "I've always felt cold when others around me felt perfectly comfortable," one woman with the disorder recalled of her upbringing.
  • Kristle Lowell, a champion trampoline gymnast, said her teammates used to call her "icy toes" because of the blue color of her feet while practicing in cold gyms.

Cool Comedy • Hot Cuisine: A Tribute to Bob Saget Raises More Than $1.2M for Scleroderma Research Foundation

Retrieved on: 
Friday, September 23, 2022

Some of the world's most talented performers generously donated their time to take to the stage to raise funds for the Scleroderma Research Foundation and to find a cure.

Key Points: 
  • Some of the world's most talented performers generously donated their time to take to the stage to raise funds for the Scleroderma Research Foundation and to find a cure.
  • Cool Comedy Hot Cuisine featured performances byHowie Mandel and Kevin Nealon, with Joel McHale leading the live auction.
  • As a longtime supporter and board member of Scleroderma Research Foundationthe country's first and leading nonprofit investor in medical research into sclerodermaBob Saget spearheaded Cool Comedy Hot Cuisine for 30 years.
  • The Scleroderma Research Foundation is focused on bringing the best minds in science together to find a cure for scleroderma.

Quoin Pharmaceuticals Establishes Patient Specific Website for Ongoing Netherton Syndrome Clinical Study

Retrieved on: 
Tuesday, September 13, 2022

ASHBURN, Va., Sept. 13, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the Company or Quoin), a specialty pharmaceutical company focused on rare and orphan diseases, has established a patient specific website: https://www.nethertonsyndromeclinicaltrials.com/ , to facilitate patient awareness and participation in its ongoing clinical study evaluating QRX003 for the treatment of Netherton Syndrome.

Key Points: 
  • ASHBURN, Va., Sept. 13, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the Company or Quoin), a specialty pharmaceutical company focused on rare and orphan diseases, has established a patient specific website: https://www.nethertonsyndromeclinicaltrials.com/ , to facilitate patient awareness and participation in its ongoing clinical study evaluating QRX003 for the treatment of Netherton Syndrome.
  • Based on discussions with the FDA, a number of different clinical endpoints will be assessed in the study.
  • We believe the establishment of this patient specific website could be an effective tool to provide information about our clinical study and facilitate patient recruitment.
  • Quoin Pharmaceuticals Ltd. is a clinical stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.

aTyr Pharma Receives FDA Fast Track Designation for Efzofitimod for Treatment of Systemic Sclerosis-Associated Interstitial Lung Disease (SSc-ILD)

Retrieved on: 
Tuesday, September 13, 2022

SAN DIEGO, Sept. 13, 2022 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that the U.S. Food and Drug Administration (FDA) has granted the company Fast Track designation for its lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis (SSc, also known as scleroderma)-associated, interstitial lung disease (ILD).

Key Points: 
  • Efzofitimod is a first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2).
  • We believe this designation further validates efzofitimod and greatly expands the market potential for this first-in-class therapeutic.
  • aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease.
  • aTyrs lead indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease.

ASK4SAM Launches New York Camp Lejeune Toxic Water Legal Injury Claims Hotline

Retrieved on: 
Friday, September 9, 2022

Silberstein, Awad, and Miklos, P.C. (ASK4SAM) has recently sponsored a toll free hotline number for New York residents who have been affected by the Camp Lejeune Toxic Water Contamination and who want to speak with an attorney at no cost about their potential claim.

Key Points: 
  • (ASK4SAM) has launched a Camp Lejeune Toxic Water Injury Claims Hotline to help residents of New York State seek compensation for themselves or their loved ones if they stationed at Camp Lejeune and were harmed by the toxic water that plagued the Marine Corps Base Camp Lejeune in Jacksonville, NC, which is in Eastern North Carolina during the qualified span of time discussed below.
  • Residents of New York State can call this number and speak to an attorney about whether they have a claim in the recently decided Camp Lejeune contaminated water supply case.
  • For New York Residents or their loved ones who were stationed at Camp Lejeune for a period of 30 days or longer are elligible to call the New York State Camp Lejeune Toxic Water Injury Claims Hotline , by calling 888-6-LEJEUNE, or by visiting ask4sam.net and clicking on the Camp Lejeune Claims option on the main page, or clicking the link in the description below to learn more about this civil claim about the contaminated drinking water on the base.
  • They have multiple office locations across New York City, and they set up the Camp Lejeune Toxic Water Injury Claim Hotline to help further streamline the fair compensation and disability benefits for those exposed to toxic materials on the military base.

Coya Therapeutics, Inc. Announces Peer-Reviewed Publication of Phase 2a Clinical Trial Data for COYA 101 in Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Thursday, September 1, 2022

Coya has an exclusive worldwide license from Houston Methodist Hospital to develop, manufacture, and commercialize COYA 101.

Key Points: 
  • Coya has an exclusive worldwide license from Houston Methodist Hospital to develop, manufacture, and commercialize COYA 101.
  • Subjects enrolled in the Phase 2a trial were initially randomized into a double-blind portion to receive active Treg cells or matching placebo every four weeks over 24 weeks.
  • The DB portion of the trial was curtailed due to COVID-19-related restrictions, and the limited number of patients enrolled made statistical analysis not feasible.
  • The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a validated rating instrument that gauges the progression of disability in patients with amyotrophic lateral sclerosis (ALS) on a 48-point scale.

Simulations Plus Releases State-of-the-Art QSP Software for Interstitial Lung Disease

Retrieved on: 
Wednesday, August 31, 2022

Simulations Plus , Inc. (Nasdaq: SLP), a leading provider of modeling and simulation software and services for pharmaceutical safety and efficacy, today announced that its DILIsym Services (DSS) division has released ILDsym version 1A, quantitative systems pharmacology (QSP) modeling software.

Key Points: 
  • Simulations Plus , Inc. (Nasdaq: SLP), a leading provider of modeling and simulation software and services for pharmaceutical safety and efficacy, today announced that its DILIsym Services (DSS) division has released ILDsym version 1A, quantitative systems pharmacology (QSP) modeling software.
  • Dr. Lisl Shoda , associate vice president, added: Interstitial lung disease significantly worsens the prognosis for patients with SSc and increases their healthcare costs by roughly 50%.
  • ILDsym and our scientific consulting expertise are positioned to support our clients in the development of novel treatments for SScILD.
  • Serving clients worldwide for more than 25 years, Simulations Plus is a leading provider in the biosimulation market providing software and consulting services supporting drug discovery, development, research, and regulatory submissions.

APIE Therapeutics Provides Evidence that Activating the Apelinergic System can Repair and Regenerate Lung Tissue Damaged by Pulmonary Fibrosis

Retrieved on: 
Tuesday, August 30, 2022

Our small molecule candidate, APT-101 has shown the potential to protect the vascular endothelium from damage and also regenerate damaged lung tissue in animal models of pulmonary fibrosis.

Key Points: 
  • Our small molecule candidate, APT-101 has shown the potential to protect the vascular endothelium from damage and also regenerate damaged lung tissue in animal models of pulmonary fibrosis.
  • The apelinergic system plays a key role in the maintenance of vascular health and function through regulation of fibrosis, cell proliferation and inflammation.
  • Further, activation of the apelinergic system through APJ has been shown to regenerate the small capillary blood vessels that are damaged by inflammation and fibrosis, which are characteristic of ILD.
  • In animal models of pulmonary fibrosis, APIE Tx has demonstrated that APT-101 diminishes the onset of fibrosis when administered before its onset and reduces collagen buildup and fibrosis when administered following pulmonary damage.

Second Annual ILD Day to Drive Awareness of Interstitial Lung Disease on Sept. 14

Retrieved on: 
Wednesday, August 24, 2022

CHICAGO, Aug. 24, 2022 /PRNewswire/ -- Nine organizations are joining forces to present the second annual ILD Day on Wednesday, Sept. 14, to drive awareness of interstitial lung disease (ILD).

Key Points: 
  • CHICAGO, Aug. 24, 2022 /PRNewswire/ -- Nine organizations are joining forces to present the second annual ILD Day on Wednesday, Sept. 14, to drive awareness of interstitial lung disease (ILD).
  • PF can be seen in many types of ILD and the damage caused by ILD can be irreversible and worsen over time.
  • "The goal of ILD Day is to expand the understanding of ILD among patients and healthcare providers, and to share resources and support for those who are living with the disease."
  • ILD Day was created to drive awareness of interstitial lung disease (ILD) in 2021 and is recognized annually on the second Wednesday in September.

Kyverna Therapeutics Names Georg Schett, M.D., and Peter A. Merkel, M.D., MPH, to Scientific Advisory Board

Retrieved on: 
Tuesday, August 23, 2022

EMERYVILLE, Calif., Aug. 23, 2022 /PRNewswire/ -- Kyverna Therapeutics ("Kyverna"), a cell therapy company engineering a new class of therapies for serious autoimmune diseases, today announced the appointment of Georg Schett, M.D., Professor and Head of the Department of Internal Medicine 3, and Vice President of Research, Friedrich-Alexander University, Erlangen-Nürnberg, Erlangen, Germany, and Peter A. Merkel, M.D., MPH, Chief of Rheumatology and Professor of Medicine and Epidemiology at the University of Pennsylvania, as Scientific Advisors.

Key Points: 
  • "We are thrilled to have Georg and Peter, two internationally recognized experts in rheumatic and autoimmune diseases, join our Scientific Advisory Board," said Dominic Borie, M.D., Ph.D., President and CEO of Kyverna.
  • Prof. Dr. Schett's interests include basic, translational, and clinical research on molecular and cellular pathogenesis of rheumatic and autoimmune diseases.
  • Kyverna Therapeutics is a cell therapy company engineering a new class of therapies for autoimmune and inflammatory diseases.
  • By offering more than one mechanism for taming autoimmunity, Kyverna is positioned to transform how autoimmune diseases are treated.