AADC

ClearPoint Neuro Congratulates its Partner PTC Therapeutics on Completion of Biologics License Application Submission to FDA for Upstaza™ as a Treatment for AADC Deficiency

Retrieved on: 
Tuesday, March 19, 2024

If approved, Upstaza™ would become the first therapy to treat AADC Deficiency in the United States.

Key Points: 
  • If approved, Upstaza™ would become the first therapy to treat AADC Deficiency in the United States.
  • “AADC Deficiency is a devastating rare pediatric movement disorder that causes significant developmental delays and autonomic symptoms starting from birth.
  • Patients with AADC deficiency are at a high risk of death in the first decade of life,” stated Jeremy Stigall, Chief Business Officer at ClearPoint Neuro.
  • Through our partnership with PTC, we are demonstrating our commitment to drive progress for the AADC Deficiency community.”

PTC Therapeutics Provides Key Regulatory Updates

Retrieved on: 
Tuesday, March 19, 2024

SOUTH PLAINFIELD, N.J., March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC expects to make the NDA resubmission by mid-year.

Key Points: 
  • SOUTH PLAINFIELD, N.J., March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
  • In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
  • PTC expects to make the NDA resubmission by mid-year.
  • The data collected to date continue to demonstrate the transformative clinical benefits the gene therapy provides to patients," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024

SOUTH PLAINFIELD, N.J., Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, at 10:30am EST/7:30am PST. Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones. Preliminary 2023 unaudited financial results and 2024 financial guidance will also be provided. The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

Key Points: 
  • Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones.
  • The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com .
  • PTC announced strategic portfolio prioritizations, which resulted in reductions in both operating expenses and headcount of approximately 25% and 30%, respectively.
  • PTC maintains all economics associated with up to $250 million in the remaining commercial sales milestones associated with Evrysdi global net sales.

ICYMI: FPF Webinar Discussed The Current State of Kids’ and Teens’ Privacy

Retrieved on: 
Tuesday, January 2, 2024

Privacy by design for kids and teens has expanded across the globe. As policymakers, advocates, and companies grapple with the ever-changing landscape of youth privacy regulation, the Future of Privacy Forum recently hosted a webinar discussing the current state of kids’ and teens’ privacy policy. The webinar explored the current frameworks that are influential worldwide, [?]

Key Points: 


Privacy by design for kids and teens has expanded across the globe. As policymakers, advocates, and companies grapple with the ever-changing landscape of youth privacy regulation, the Future of Privacy Forum recently hosted a webinar discussing the current state of kids’ and teens’ privacy policy. The webinar explored the current frameworks that are influential worldwide, [?]

Five Big Questions (and Zero Predictions) for the U.S. State Privacy Landscape in 2024

Retrieved on: 
Tuesday, January 2, 2024

Entering 2024, the United States now stands alone as the sole G20 nation without a comprehensive, national framework governing the collection and use of personal data. With bipartisan efforts to enact federal privacy legislation once again languishing in Congress, state-level activity on privacy dramatically accelerated in 2023. As the dust from this year settles, we [?]

Key Points: 


Entering 2024, the United States now stands alone as the sole G20 nation without a comprehensive, national framework governing the collection and use of personal data. With bipartisan efforts to enact federal privacy legislation once again languishing in Congress, state-level activity on privacy dramatically accelerated in 2023. As the dust from this year settles, we [?]

eAssist Partners with Henry Schein and Other Dental Industry Experts to Launch 2024 Give Yourself a Raise Tour

Retrieved on: 
Monday, December 11, 2023

SALT LAKE CITY, Dec. 11, 2023 /PRNewswire/ -- eAssist Dental Solutions ( www.dentalbilling.com ), the nation's leading dental billing company, again partners with Henry Schein and other dental industry experts in offering a revenue cycle management seminar to help dentists and practice owners increase profits.

Key Points: 
  • SALT LAKE CITY, Dec. 11, 2023 /PRNewswire/ -- eAssist Dental Solutions ( www.dentalbilling.com ), the nation's leading dental billing company, again partners with Henry Schein and other dental industry experts in offering a revenue cycle management seminar to help dentists and practice owners increase profits.
  • "We joined with Henry Schein in launching the first Give Yourself a Raise Tour this year, and we're excited to help ramp up the nationwide 2024 Tour," said eAssist CEO and co-founder James Anderson.
  • In August 2023, Henry Schein inaugurated the first Give Yourself a Raise Tour, which included eAssist and other respected dental industry partners.
  • The 2024 Tour will again feature the following key industry experts.

eAssist to Present at Flagship Event by American Academy of Implant Dentistry (AAID)

Retrieved on: 
Thursday, November 2, 2023

The event, themed "Trends in Transformation," will be held November 1-4 at Caesar's Palace in Las Vegas.

Key Points: 
  • The event, themed "Trends in Transformation," will be held November 1-4 at Caesar's Palace in Las Vegas.
  • Dr. Jim DiMarino is the Chief Executive Officer at Practice Booster, an eAssist Dental Solutions company.
  • He completed a General Practice Residency, became a solopreneur practicing general dentistry, all while researching and implementing advanced dental technologies.
  • She has over 30 years of experience in dentistry and has been designated a Leader in Dental Consulting by Dentistry Today from 2007 to present.

Vigil Neuroscience Appoints Christopher J. Silber, M.D. as Chief Medical Officer

Retrieved on: 
Monday, August 7, 2023

as Chief Medical Officer.

Key Points: 
  • as Chief Medical Officer.
  • Dr. Silber brings over 30 years of biopharmaceutical industry experience and a track record of clinical and regulatory success developing novel therapeutics in neuroscience.
  • “We are thrilled to welcome Dr. Silber to Vigil as our new Chief Medical Officer at this pivotal time as we advance VGL101 through Phase 2 clinical development and work towards moving our small molecule program into the clinic later this year,” said Ivana Magovčević-Liebisch, Ph.D., J.D., President and Chief Executive Officer of Vigil.
  • “Vigil’s precision-based development approach targeting microglia represents a new frontier in the treatment of neurodegenerative diseases, which remains an area of high unmet medical need,” said Dr. Silber.