University of California, San Francisco Fetal Treatment Center

Achieve Life Sciences Announces Presentation of Cytisinicline E-Cigarette Cessation (ORCA-V1) Results at Society for Research on Nicotine and Tobacco Europe (SRNT-E) Annual Meeting

Retrieved on: 
Monday, September 11, 2023

The positive topline results from the ORCA-V1 trial, announced earlier this year, will be presented, as well as additional findings.

Key Points: 
  • The positive topline results from the ORCA-V1 trial, announced earlier this year, will be presented, as well as additional findings.
  • Additional analyses showed that vaping abstinence with cytisinicline treatment started by the second week of treatment, with the odds of vaping cessation improving throughout the 12-week treatment period.
  • During the Week 12-16 follow-up period, subjects treated with cytisinicline continued to demonstrate 2.0 times higher odds of vaping cessation compared to placebo.
  • The content is the sole responsibility of the authors and does not necessarily represent the official views of the NIH.

Thrive Health IV Centers Expand in Key Markets and Open Research Clinics

Retrieved on: 
Tuesday, September 5, 2023

BEVERLY HILLS, Calif., Sept. 5, 2023 /PRNewswire-PRWeb/ -- Thrive Health IV, founded by renowned board certified oculoplastic surgeon, Raymond Douglas, MD, PhD, announced today the opening of three new dedicated treatment centers of excellence in Miami, Florida, Des Moines, Iowa and Chicago, Illinois for Thyroid Eye Disease [TED] and other rare ophthalmic conditions.

Key Points: 
  • Thrive Health IV leverages cutting edge science and exceptional concierge care in the growing category of medical biologics and new disease modalities.
  • Thrive Health IV is also launching a new research division and operating clinical trials at all sites, propelling Thrive Health IV to the forefront of all potential therapies for auto-immune thyroid related eye disease and other rare ophthalmic conditions.
  • Additional Thrive Health IV locations with research and clinical trial operations are set to open in two new states in three additional markets as a next phase before 2024.
  • Thrive Health IV centers of excellence, also offers ongoing patient care management that includes monitoring blood sugar and complimentary audiology testing.

Amicus Therapeutics Announces Presentation and Posters at the SSIEM Annual Symposium 2023

Retrieved on: 
Tuesday, August 29, 2023

PHILADELPHIA, Aug. 29, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that one oral presentation and five posters across its development programs will be included at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 , being held August 29 – September 1, 2023 in Jerusalem, Israel.

Key Points: 
  • PHILADELPHIA, Aug. 29, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that one oral presentation and five posters across its development programs will be included at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 , being held August 29 – September 1, 2023 in Jerusalem, Israel.
  • Abstract Title: Multiorgan involvement in females with Fabry disease: results from two phase III trials and the followME registry (Poster #B169)
    Abstract Title: Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials (Poster #B20)
    Presenter: Ozlem Goker-Alpan, MD, Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, U.S.A

Stoke Therapeutics Announces Positive New Safety & Efficacy Data from Patients Treated with STK-001 in the Phase 1/2a Studies (MONARCH & ADMIRAL) and the SWALLOWTAIL Open-Label Extension (OLE) Study in Children and Adolescents with Dravet Syndrome

Retrieved on: 
Tuesday, July 25, 2023

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced positive new safety and efficacy data from patients treated with STK-001 in the two ongoing Phase 1/2a studies (MONARCH and ADMIRAL) and the SWALLOWTAIL open-label extension (OLE) study in children and adolescents with Dravet syndrome.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced positive new safety and efficacy data from patients treated with STK-001 in the two ongoing Phase 1/2a studies (MONARCH and ADMIRAL) and the SWALLOWTAIL open-label extension (OLE) study in children and adolescents with Dravet syndrome.
  • These new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001.
  • The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population.
  • “Together these data support the potential for STK-001 to address the underlying cause of Dravet syndrome by treating both seizures and the cognitive and behavioral issues that make this disease so complex and devastating.

REGENXBIO Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RGX-121 Gene Therapy for Hunter Syndrome

Retrieved on: 
Tuesday, May 23, 2023

"I am encouraged by the FDA's recognition of the potential of a gene therapy like RGX-121 to improve the quality of life for boys with severe Hunter syndrome and the urgent need for new treatment options."

Key Points: 
  • "I am encouraged by the FDA's recognition of the potential of a gene therapy like RGX-121 to improve the quality of life for boys with severe Hunter syndrome and the urgent need for new treatment options."
  • "The expedited development of new treatment options like RGX-121 are critical to families facing this devastating diagnosis."
  • In addition, improvements in neurodevelopmental and daily activity skill acquisition were observed up to three years after RGX-121 administration.
  • A Phase I/II trial of RGX-121 for the treatment of pediatric patients with MPS II over the age of five years old is also ongoing.

Dr. Scott D. Flamm, Former Section Head of Cardiovascular Imaging at Cleveland Clinic, Joins DocPanel

Retrieved on: 
Monday, May 1, 2023

Formerly with Cleveland Clinic, Dr. Flamm served as Head of Cardiovascular Imaging with joint appointments in the Imaging Institute, Heart, Vascular, and Thoracic Institute, and the Pediatric Institute (2006-2023).

Key Points: 
  • Formerly with Cleveland Clinic, Dr. Flamm served as Head of Cardiovascular Imaging with joint appointments in the Imaging Institute, Heart, Vascular, and Thoracic Institute, and the Pediatric Institute (2006-2023).
  • "DocPanel and I share a mutual mission to make subspecialized cardiac imaging routine care that can be accessed by all," says Dr. Flamm.
  • Through the DocPanel platform, healthcare facilities can access Dr. Flamm for dedicated cardiothoracic imaging coverage, protocol assistance, and program development support.
  • Dr. Flamm completed fellowship training in Cardiovascular Imaging and MRI at the University of California San Francisco under Dr. Charles Higgins.

Renowned NYC Orthopedic Surgeon, Dr. Steven Struhl, Performs Successful Surgery on Broadway Star

Retrieved on: 
Thursday, March 30, 2023

NEW YORK, March 30, 2023 /PRNewswire-PRWeb/ -- Dr. Steven Struhl is a well-respected, board-certified orthopedic surgeon and sports medicine expert. He is a leading NYC shoulder specialist, providing optimal care and repair through arthroscopic procedures. One of Dr. Struhl's patients, stage actor and dancer Ben Bogan, seen on Broadway in FROZEN and JERSEY BOYS, recently suffered from a serious shoulder dislocation. With shoulder injuries or problems, getting an accurate diagnosis and immediate care is crucial so elite performers and athletes can get back to normal. Many dancers and talented theater stars, like Mr. Bogan, have trusted Dr. Struhl as their preferred shoulder and knee specialist. When major shoulder injuries are addressed promptly, they can be repaired quickly and effectively through Dr. Steven Struhl's arthroscopic surgical skill. The excellent recovery and rehab time that Mr. Bogan experienced by following Dr. Struhl's protocols allowed him to return to the stage much earlier than expected.

Key Points: 
  • Broadway performer, Ben Bogen recently suffered from a serious shoulder dislocation which Dr. Steven Struhl helped to repair.
  • NEW YORK, March 30, 2023 /PRNewswire-PRWeb/ -- Dr. Steven Struhl is a well-respected, board-certified orthopedic surgeon and sports medicine expert.
  • When major shoulder injuries are addressed promptly, they can be repaired quickly and effectively through Dr. Steven Struhl's arthroscopic surgical skill.
  • Dr. Steven Struhl is board certified in both orthopedic surgery and sports medicine/arthroscopic surgery and has been in private practice for many years.

National Lyme Clinical Trials Network Gains Its First West Coast Center, Thanks to $1M+ Donation from Bay Area Lyme Foundation

Retrieved on: 
Thursday, March 9, 2023

This new center will become the first West Coast site of the Lyme Clinical Trials Network .

Key Points: 
  • This new center will become the first West Coast site of the Lyme Clinical Trials Network .
  • “The founding of the UCSF Lyme Clinical Trials Center provides a unique opportunity for Lyme patients to participate in the next generation of therapeutic trials to combat this devastating disease,” said Charles Chiu, MD, PhD, of the University of California San Francisco who will lead the UCSF Lyme Clinical Trials Center.
  • “The Lyme Clinical Trials Network was created to help bring treatments and hope to Lyme disease patients and their families,” said Alexandra Cohen, president of the Steven & Alexandra Cohen Foundation.
  • His team will be evaluating and treating patients for case registry and pilot studies, and publishing in conjunction with other Lyme Clinical Trials Network sites.

Achieve Life Sciences Announces Last Subject Last Visit Completed in Phase 2 ORCA-V1 Trial of Cytisinicline for e-Cigarette Cessation

Retrieved on: 
Wednesday, March 8, 2023

“We are excited to have reached this important milestone in the ORCA-V1 trial, bringing us one step closer to understanding the cessation potential of cytisinicline in users of nicotine e-cigarettes who desire to quit,” stated John Bencich, Chief Executive Officer of Achieve.

Key Points: 
  • “We are excited to have reached this important milestone in the ORCA-V1 trial, bringing us one step closer to understanding the cessation potential of cytisinicline in users of nicotine e-cigarettes who desire to quit,” stated John Bencich, Chief Executive Officer of Achieve.
  • Dr. Nancy Rigotti, Professor of Medicine at Harvard Medical School and Director, Tobacco Research and Treatment Center, Massachusetts General Hospital is the primary investigator for the ORCA-V1 trial.
  • For more information on cytisinicline or the ORCA-V1 trial, please visit www.achievelifesciences.com .
  • The content is the sole responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Achieve Life Sciences Announces Presentation of the Phase 3 ORCA-2 Trial of Cytisinicline at Society for Research on Nicotine and Tobacco (SRNT) Annual Meeting

Retrieved on: 
Friday, March 3, 2023

The presentation will be given by ORCA-2 Principal Investigator, Dr. Nancy Rigotti, Professor of Medicine at Harvard Medical School and Director, Tobacco Research and Treatment Center, Massachusetts General Hospital.

Key Points: 
  • The presentation will be given by ORCA-2 Principal Investigator, Dr. Nancy Rigotti, Professor of Medicine at Harvard Medical School and Director, Tobacco Research and Treatment Center, Massachusetts General Hospital.
  • Subjects who received either 6 or 12-weeks of cytisinicline treatment experienced consistently higher rates of abstinence.
  • The Company continues to expect topline data results for both trials to be announced in the second quarter of 2023.
  • To learn more about SRNT or the SRNT Annual Meeting, please visit www.srnt.org .