The PennZone

  • Home
  • Business
  • Health
  • Education
  • Construction
  • Financial
  • Legal
  • Entertainment
  • Books

Cure SMA Announces New Funding For Network Of Prominent SMA Clinical Research And Treatment Centers
The PennZone/10070663

Trending...
  • VIP Vacations Recognized as a 2024 Top Selling Travel Agency by the Jamaica Tourism Board
  • Pyro Marketing Opens New Digital Marketing Company to Power Growth for Fitness and Ecommerce Brands
  • Assent Expands Executive Team to Accelerate Global Growth & Innovation
Cure SMA
The Pediatric Neuromuscular Clinical Research Network Will Continue to Drive Treatment Development Under Cure SMA's Funding

CHICAGO - PennZone -- In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is pleased to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide $1.2 million to this Network of highly skilled clinical trial investigators, clinical evaluators, clinical coordinators, statisticians, and data management personnel.

In recent years, there have been many important medical breakthroughs, offering new and effective SMA treatment options. The PNCRN has been involved in the seminal clinical trials that have led to the FDA approvals of anti-sense oligonucleotides, gene transfer strategies, and other breakthrough treatments that have changed forever the natural course of the disease. Their work continues to develop and refine more sensitive outcome measures, conduct ongoing clinical trials, accelerate newborn screening programs, and identify promising new treatment approaches.

"In these unprecedented times, we see the investment in the PNCRN as critical in our mission to advance treatment and care," says Kenneth Hobby, President, Cure SMA, the leading national organization dedicated to finding effective treatments, care protocols and, ultimately, a cure for SMA. "We are eager to build on the past accomplishments of the Network, made possible by the generous and continuous sponsorship from the SMA Foundation."

Originally funded by the SMA Foundation in 2004, the PNCRN established a team of SMA clinical trial experts that have integrated clinical research, education, and care to achieve the best possible clinical trial outcomes. Cure SMA has collaborated with the SMA Foundation as co-sponsor of the PNCRN since 2018.

"The SMA Foundation has built and supported this Network for the past 15 years to establish a group of multidisciplinary SMA expert centers that support clinical trials," said Loren A. Eng, President, SMA Foundation. "Through the PNCRN, the Foundation has supported key clinical research studies, such as the SMA Natural History Study and the development of key SMA outcome measures that currently are accepted and used globally. The Natural History of SMA in the modern era was clearly defined by the Network and has been used as a key benchmark to assess safety and effectiveness of potential therapies, and ultimately to obtain recent treatment approvals."

More on The PennZone
  • ICAST 2025: The Crystal-Clear Revolution Begins at Booth #3233
  • Brindle Pet Supplies Now Carries Badlands Ranch Dog Food in Canada
  • $10 Million Allocated to Establish Crypto Treasury Focused on High Value Ethereum (ETH) & Bitcoin (BTC) as Long-Term Holdings for Cybersecurity Leader
  • Cummings Graduate Institute for Behavioral Health Studies Celebrates New DBH Graduates
  • Together We Dance Foundation and Newtown Athletic Club Unite for Inclusive Swim Party

"Since the advent of effective SMA treatments, the recognition and description of new SMA phenotypes in treated patients and the durability of the approved treatments have emerged as critical issues, making the continuing work to understand the disease mechanisms and phenotype-genotype correlations that much more important," says Darryl De Vivo, M.D., Director of the PNCRN and Professor of Neurology and Pediatrics, Columbia University Irving Medical Center, New York City. "Cure SMA's leadership in SMA research, and its new support of the PNCRN, will allow us to sustain this ground-breaking collaboration in SMA research and clinical care, especially as we expand the number of effective treatments for SMA and pursue the ultimate vision of a cure."

Six sites comprise the PNCRN for SMA, including Boston Children's Hospital, Boston, MA; Children's Hospital of Philadelphia, Philadelphia, PA; Columbia University Irving Medical Center, New York, NY; Nemours Children's Health System, Orlando FL; Stanford University, Palo Alto, CA; and the data coordinating center at the University of Rochester, Rochester, NY.

These clinical research and treatment sites have also now been integrated into the established Cure SMA Care Center Network, which will lead to real-world evidence that increases access to approved treatments and improves care for individuals and families with SMA.

About SMA

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that robs an individual of their ability to walk, swallow, and—in the most severe cases—even breathe. SMA is the leading genetic cause of death for infants. Symptoms can surface within the first 6 months of life (Type 1, the most severe and common), during the toddler years (Types 2 and 3), or in adulthood (Type 4, the least common form). SMA affects 1 in 11,000 births in the United States each year, and approximately 1 in 50 Americans is a genetic carrier. The good news is that there are now truly effective FDA-approved treatments for SMA that make it possible for individuals with SMA to achieve developmental milestones and live full and productive lives.

More on The PennZone
  • $100 to $200 Million Equity Agreement with Top Digital Advisor Bitwise to Power Major Digital Asset Initiative for Bitcoin and Solana: OFA Group
  • New Collaboration Launches Corporate ESG Solution for Responsible Decommissioning and Transparent Reporting
  • Beyond Spots & Dots Wins Creative Awards for Marketing Initiatives
  • SlickCashLoan Launches Free Loan Calculator to Help You Plan Monthly Payments
  • Solution for Broken Honda Keys: All City Locksmith Launches Durable, Break-Resistant Replacements

About Cure SMA

Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA). Since 1984, Cure SMA has grown to be the largest network of individuals, families, clinicians, and research scientists working together to advance SMA research, support affected individuals/caregivers, and educate public and professional communities about SMA. The organization has directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA, led to breakthroughs in treatment and care, and provided individuals and families the support they need today. For more information, visit www.cureSMA.org.

About SMA Foundation

Founded in 2003, the SMA Foundation is a nonprofit organization dedicated to accelerating the development of treatments for SMA through targeted funding of clinical research and novel drug development efforts. Since its inception, the Foundation has awarded ~$150 million for SMA research. In addition, the Foundation is committed to raising awareness and generating support for increased research efforts in SMA among the leaders of industry and government. When the SMA Foundation was founded, there were no treatments for SMA available. Today there are two approved treatments and several more in clinical and preclinical development. For more information, visit the SMA Foundation website at http://www.smafoundation.org.

Contact
Leslie Humbel
***@curesma.org


Source: Cure SMA
Filed Under: Health

Show All News | Report Violation

0 Comments
1000 characters max.

Latest on The PennZone
  • Smart Resnse Unveils Smart Resnse(SRMS) Token-Powered AI Orchestration Platform to Revolutionize Multi-Billion Dollar Market
  • Josh and Heidi Follow Up the Much Anticipated and Successful Launch of the "Spreading the Good BUZZ" Podcast with a Personal Request
  • Revolutionary Blockchain Platform Okh Finance Announces Okh Finance(OKKH) Token Launch to Transform Global Asset Leasing Market
  • Cover Girl Finalist Teisha Mechetti Questions Legitimacy of Inked Originals Competition, Demands Transparency
  • Franchise-Ready: Wise Business Plans Partners with Leading Franchisors to Support New Franchisees
  • VIP Vacations Recognized as a 2024 Top Selling Travel Agency by the Jamaica Tourism Board
  • Easton & Easton, LLP Files Suit Against The Dwelling Place Anaheim & Vineyard USA Over Abuse Allegations
  • AI Visibility: The Key to Beating Google's AI Overviews and Regaining Traffic
  • Stuck Doing Math or Figuring Out Life's Numbers? Calculator.now Makes It Stupidly Simple
  • CLASS ACTION REMINDER: Berger Montague Advises Hims & Hers Health Inc. (NYSE: HIMS) Investors to Inquire About a Securities Fraud Lawsuit by August 25, 2025
  • Colbert Packaging Announces WBENC Recognition
  • SHI Cryogenics Group Launches Highest-Capacity Single-Stage Cryocoolers to Date
  • DivX Empowers Media Enthusiasts with Free Expert Guides for Advanced MP4 Management
  • Federated Hermes Premier Municipal Income Fund declares dividend
  • Assent Expands Executive Team to Accelerate Global Growth & Innovation
  • The World's Largest Green Economic Revolution Emerges as Nature, Tech, and Finance Converge
  • Vinnetwork Unveils Decentralized AI Platform with Vinnetwork(VIN) Token to Challenge Tech Giants' Data Monopoly
  • Centennial Flyers to Become Colorado's First Launch Customer for All-Electric B23 Energic Aircraft
  • Pyro Marketing Opens New Digital Marketing Company to Power Growth for Fitness and Ecommerce Brands
  • Dr. John Salerno of Salerno Wellness Introduces Their New Full Body Capsule for Advanced LED Light Therapy Patient Treatments

Popular on PennZone

  • INVESTIGATION ALERT: Berger Montague PC Investigates Coinbase Global, Inc.'s Board Of Directors For Breach of Fiduciary Duties (NASDAQ: COIN) - 112
  • IRF Builders Forum Brings Global Leaders to Washington, D.C. to Advance Religious Freedom Through Cooperative Engagement
  • New Bethany Acquires Former Diocesan Headquarters to Expand Services
  • Bethany Nikitenko Honored By Legal Intelligencer with Power Player Award
  • JOSHWAY Launches Children's Book, "JOSHWAY and the Power of Resilience"
  • OPRAH.COM Featured Award-Winning Novel AS FAR AS YOU GO BEFORE YOU HAVE TO COME BACK now Available as Audiobook
  • Joyce Carol Oates Returns to Hard Case Crime With DOUBLE TROUBLE
  • Jeopardy!'s Ken Jennings Headlines National Mensa Event
  • KeysCaribbean Vacation Home Rentals Offers Last-Minute Booking Discount of 15 Percent
  • Coming Soon: The Cottages at Powell Park Offer a Rare Blend of Charm, Convenience, and Community in Raleigh

Similar on PennZone

  • Fastest Growing Staffings Firms
  • Cummings Graduate Institute for Behavioral Health Studies Celebrates New DBH Graduates
  • Pyro Marketing Launches New Website to Accelerate Growth for Fitness Brands
  • The Citizens Commission on Human Rights Annual Purple Heart Day Event will be Hosted at the Historic Fort Harrison
  • Psychedelics for Vets? CCHR Cites History of Exploitation and Failed Science
  • The Blue Luna Encourages Local Schools to Take Steps to Enhance Safety for Students and Staff
  • Josh and Heidi Follow Up the Much Anticipated and Successful Launch of the "Spreading the Good BUZZ" Podcast with a Personal Request
  • Stuck Doing Math or Figuring Out Life's Numbers? Calculator.now Makes It Stupidly Simple
  • Pyro Marketing Opens New Digital Marketing Company to Power Growth for Fitness and Ecommerce Brands
  • Dr. John Salerno of Salerno Wellness Introduces Their New Full Body Capsule for Advanced LED Light Therapy Patient Treatments
Copyright © The PennZone | Theme: OMag by LilyTurf Themes
  • Contribute
  • Privacy Policy
  • Terms of Service
  • Contact Us