Adrenal insufficiency

Ellodi Pharmaceuticals Announces FLUTE-2 Data to be Presented at DDW 2024 with APT-1011 in Patients with Eosinophilic Esophagitis

Retrieved on: 
Dienstag, Mai 21, 2024

Ellodi Pharmaceuticals, a gastroenterology-focused specialty pharmaceutical company, today announced an oral presentation by Professor Evan Dellon M.D., M.P.H, at the Digestive Disease Week (DDW) 2024 Annual Scientific Meeting.

Key Points: 
  • Ellodi Pharmaceuticals, a gastroenterology-focused specialty pharmaceutical company, today announced an oral presentation by Professor Evan Dellon M.D., M.P.H, at the Digestive Disease Week (DDW) 2024 Annual Scientific Meeting.
  • The presentation will highlight encouraging results of APT-1011 (fluticasone propionate orally disintegrating tablet) in the FLUTE-2 Phase 3 clinical study in Eosinophilic Esophagitis (EoE).
  • Dr. Dellon, who served as the Primary Investigator for FLUTE-2, will present the data on Tuesday, May 21 at 8am ET.
  • The results of the FLUTE-2 study indicate that APT-1011 could provide symptomatic and histologic benefits and help to fulfill the unmet need in patients with EoE.

Quince Therapeutics Provides Business Update and Reports First Quarter 2024 Financial Results

Retrieved on: 
Montag, Mai 13, 2024

Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, today provided an update on the company’s development pipeline and reported financial results for the first quarter ended March 31, 2024.

Key Points: 
  • Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, today provided an update on the company’s development pipeline and reported financial results for the first quarter ended March 31, 2024.
  • Commenced NEAT study site initiation and activation activities throughout the U.S., U.K., and European Union as NEAT enrollment remains on track to begin in the second quarter of 2024.
  • Reported cash, cash equivalents, and short-term investments of $67.8 million for the first quarter ended March 31, 2024.
  • Reported a net loss of $11.1 million, or a loss of $0.26 per basic and diluted share, for the quarter ended March 31, 2024.

Polarean’s Xenon MRI to be Featured at Upcoming ATS 2024 Conference

Retrieved on: 
Mittwoch, Mai 8, 2024

The summit is scheduled to take place on May 17th-18th in San Diego, CA, at the Manchester Grand Hyatt San Diego.

Key Points: 
  • The summit is scheduled to take place on May 17th-18th in San Diego, CA, at the Manchester Grand Hyatt San Diego.
  • The Respiratory Innovation Summit, a premier event in the field of pulmonary medicine, unites innovators, investors, clinicians, and advocacy groups.
  • After the dynamic discussions of the Respiratory Innovation Summit, Polarean eagerly anticipates further collaboration within the pulmonary medicine community at the 2024 ATS International Conference, held from May 19th to 22nd.
  • Christopher von Jako, PhD, CEO of Polarean, said: “The Respiratory Innovation Summit and the ATS annual conference serve as invaluable venues to spotlight our Xenon MRI platform, especially to those enthusiastic about fostering collaborative partnerships.

Corcept Announces Positive Results From Open-Label Portion of Pivotal Phase 3 GRACE Trial in Patients With Cushing’s Syndrome

Retrieved on: 
Montag, April 22, 2024

In the “open-label” phase, 152 patients with Cushing’s syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks.

Key Points: 
  • In the “open-label” phase, 152 patients with Cushing’s syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks.
  • GRACE’s primary endpoint is maintenance of blood pressure control in the “randomized withdrawal” phase, with maintenance of glycemic control as the key secondary endpoint.
  • Other key secondary and exploratory endpoints in the randomized withdrawal phase include changes in weight, waist circumference, cognitive impairment and Cushing’s Quality of Life score.
  • Patients in the open-label phase exhibited clinically meaningful and statistically significant improvements in hypertension, hyperglycemia and other key secondary and exploratory endpoints.

Neurocrine Biosciences Launches WHAT THE C@H?! Educational Initiative to Support Congenital Adrenal Hyperplasia Community

Retrieved on: 
Mittwoch, April 24, 2024

provides a platform for the congenital adrenal hyperplasia (CAH) community to find educational information, share experiences and learn about current research.

Key Points: 
  • provides a platform for the congenital adrenal hyperplasia (CAH) community to find educational information, share experiences and learn about current research.
  • SAN DIEGO, April 24, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the launch of WHAT THE C@H?!
  • , an educational initiative that aims to close the gap in the need for helpful information about congenital adrenal hyperplasia (CAH) and acknowledges the frustrations and challenges experienced by the community in managing the condition.
  • "We hope this educational initiative, which was informed by insights from the community, is helpful to those navigating and managing this difficult condition."

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Montag, April 8, 2024

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Montag, April 8, 2024

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

OrthoPediatrics Corp. Launches New RESPONSE Rib and Pelvic Fixation System for EOS Scoliosis

Retrieved on: 
Montag, März 4, 2024

WARSAW, Ind., March 04, 2024 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS a company focused exclusively on advancing the field of pediatric orthopedics, today announced it has launched a new RESPONSE™ Rib and Pelvic Fixation system to treat children with Early Onset Scoliosis (“EOS”).

Key Points: 
  • WARSAW, Ind., March 04, 2024 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS a company focused exclusively on advancing the field of pediatric orthopedics, today announced it has launched a new RESPONSE™ Rib and Pelvic Fixation system to treat children with Early Onset Scoliosis (“EOS”).
  • The new addition to the RESPONSE™ family includes implants and instruments for rib and pelvic fixation, and associated devices to connect the fixation points.
  • As an adjunct to the foundational RESPONSE™ Scoliosis System, the Rib and Pelvic system is compatible with 5.5/6.0 and 4.5/5.0 RESPONSE™ Systems, allowing surgeons to treat the entire spectrum of patients with scoliosis.
  • Greg Odle, President of the OrthoPediatrics’ Scoliosis Division echoed his excitement for the launch of this new system, saying “We are excited to enter the Early Onset Scoliosis treatment market with the RESPONSE™ Rib & Pelvic System.

The Wyss Foundation Provides $5 Million in Funding for Children's Hospital of Philadelphia's Wyss/Campbell Center for Thoracic Insufficiency Syndrome

Retrieved on: 
Donnerstag, Februar 22, 2024

PHILADELPHIA, Feb. 22, 2024 /PRNewswire/ -- Children's Hospital of Philadelphia (CHOP) is thrilled to announce a gift of $5 million from the Wyss Foundation to support innovations that improve the lives of patients with thoracic insufficiency syndrome (TIS). An initial gift from Hansjörg Wyss, philanthropist and chairman of the Wyss Foundation, was made to CHOP in 2019 and honored the legacy of the late Dr. Robert Campbell, a world-renowned CHOP pediatric orthopaedic surgeon. Now, additional funding has renamed and established the Wyss/Campbell Center for Thoracic Insufficiency Syndrome, one of CHOP's frontier programs, a trailblazing group of initiatives pioneering new advances in children's health. 

Key Points: 
  • Now, additional funding has renamed and established the Wyss/Campbell Center for Thoracic Insufficiency Syndrome , one of CHOP's frontier programs, a trailblazing group of initiatives pioneering new advances in children's health.
  • The $5 million gift will ensure the Center, its clinical team, researchers and patients have the resources needed to thrive by amplifying studies, tools and education.
  • The Center will be named Wyss/Campbell Center for Thoracic Insufficiency Syndrome in perpetuity.
  • The Wyss Foundation is honored to support the team working to improve the quality of life for children living with TIS," said Molly McUsic, President of the Wyss Foundation.

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Retrieved on: 
Sonntag, Februar 4, 2024

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Key Points: 


Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive