PPMD

Duchenne UK and Parent Project Muscular Dystrophy Award $500,000 to Evaluate Safety and Tolerability of Muscle Progenitor Cells in Phase 1 Trial

Retrieved on: 
화요일, 4월 23, 2024
University, Cell, Hope, Safety, DMD, Research, Dystrophin, Entrepreneurship, Patient, PPMD, Translational medicine, Duchenne muscular dystrophy, Clinical trial, Therapy, Organization, Injection, CGMP, Pharmaceutical industry

The grant, totaling $500,000, will support Dr. Kang and his co-investigators in evaluating the safety and tolerability of muscle progenitor cells for Duchenne muscular dystrophy.

Key Points: 
  • The grant, totaling $500,000, will support Dr. Kang and his co-investigators in evaluating the safety and tolerability of muscle progenitor cells for Duchenne muscular dystrophy.
  • Dr. Kang's project builds on previous research, utilizing muscle progenitor cells, known as MyoPAXon, developed by Dr. Rita Perlingeiro and her team at the University of Minnesota.
  • This Phase 1 trial led by Dr. Kang's team will provide valuable insights into the safety and feasibility of transplanting MyoPAXon cells.
  • Emily Reuben, Co-Founder and CEO of Duchenne UK, explains:
    "Duchenne UK has supported Dr. Perlingeiro's work on developing muscle progenitor cells for a number of years.

Parent Project Muscular Dystrophy Celebrates 30th Anniversary with Launch of PPMD Together Meeting Series in Cincinnati, Ohio

Retrieved on: 
수요일, 4월 17, 2024
City, Hope, PTC Therapeutics, Today, Series, Empowerment, Becker muscular dystrophy, Parent, Research, Human, Catalyst Pharmaceuticals, Policy, Family, PPMD, Sarepta Therapeutics, Diagnosis, Duchenne muscular dystrophy, Grandparent, Therapy

CINCINNATI, April 17, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), proudly marks its 30th anniversary at the launch of the PPMD Together meeting series, taking place April 19-20, 2024, in Cincinnati, Ohio. Founded three decades ago by a small group of passionate parents and grandparents within the Duchenne community, PPMD has grown into a beacon of hope, driven by the unwavering dedication of parents, families, and individuals living with Duchenne and Becker.

Key Points: 
  • CINCINNATI, April 17, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , proudly marks its 30th anniversary at the launch of the PPMD Together meeting series, taking place April 19-20, 2024, in Cincinnati, Ohio.
  • In April 1994, Cincinnati served as the birthplace of PPMD, where a handful of determined families joined forces to confront the challenges posed by Duchenne.
  • PPMD selected the location and date of the first PPMD Together meeting to celebrate the 30th anniversary of the organization in its hometown of Cincinnati, April 19-20, 2024.
  • Sponsorship for the PPMD Together meeting has been provided by Catalyst Pharmaceuticals, Edgewise Therapeutics, NS Pharma, Pfizer, PTC Therapeutics, and Sarepta Therapeutics in accordance with PPMD's Corporate Relations Policy .

Parent Project Muscular Dystrophy Launches First State-Specific Duchenne Advocacy Day in Illinois

Retrieved on: 
월요일, 4월 15, 2024
Policy, Becker muscular dystrophy, Conference, Research, Duchenne muscular dystrophy, Disease, PPMD, Becker

SPRINGFIELD, Ill., April 15, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , is proud to announce the launch of its first state-specific Duchenne Advocacy Day in Illinois.

Key Points: 
  • SPRINGFIELD, Ill., April 15, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , is proud to announce the launch of its first state-specific Duchenne Advocacy Day in Illinois.
  • The Advocacy Day in Illinois will focus on championing key legislative measures, including:
    HR 636 (Rep. Robyn Gabel)/SR 820 (Sen. Laura Fine), designating September 7, 2024, as Duchenne Muscular Dystrophy Awareness Day in the State of Illinois.
  • "The launch of this pilot project reflects our commitment to advocating for individuals and families affected by Duchenne and Becker at every level," said Lauren Stanford, Director of Advocacy at Parent Project Muscular Dystrophy.
  • For more information about Parent Project Muscular Dystrophy and its advocacy initiatives, please visit PPMD's website .

Italfarmaco Receives FDA Approval for Duvyzat™ (givinostat) in Duchenne Muscular Dystrophy

Retrieved on: 
금요일, 3월 22, 2024
Research, FDA, Genetics, Clinical Trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, HDAC, ITF, MAA, The Lancet, Caregiver, Disease, Magnetic resonance imaging, PPMD, Patient, Rehabilitation, Lancet Neurology, University, EMA, Doctor of Philosophy, MD, Duchenne muscular dystrophy, Pediatrics, Therapy, NSAA, DMD, Food, Pharmaceutical industry

“We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.

Key Points: 
  • “We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.
  • The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”
    The approval is based on the results of the pivotal multicentre, randomised, double-blind, placebo-controlled phase 3 EPIDYS trial ( NCT02851797 ).
  • Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary, ITF Therapeutics LLC.
  • Italfarmaco has a global presence and is also working with other regulatory agencies.

PPMD Applauds FDA for Approval of DUVYZAT™ (Givinostat) for Duchenne Patients Ages Six Years and Older

Retrieved on: 
금요일, 3월 22, 2024
HDAC, ITF, PPMD, Inflammation, Patient, Knowledge, Research, Policy, Duchenne muscular dystrophy, Therapy, Atrophy, FDA, Becker muscular dystrophy, Food, Pharmaceutical industry

DUVYZAT™ marks a significant milestone as the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant.

Key Points: 
  • DUVYZAT™ marks a significant milestone as the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant.
  • "PPMD is thrilled that the FDA has approved DUVYZAT™, an HDAC inhibitor therapy that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
  • Today's approval expands the therapeutic options for individuals with Duchenne," said PPMD Founding President and CEO Pat Furlong.
  • PPMD also takes a lead role in advocating for national newborn screening efforts for Duchenne, Becker, and carrier identification, as well as policy efforts around access and reimbursement.

Capricor Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
목요일, 2월 29, 2024
Vaccine, Project, Webcast, Exosome, Protein, Poster, Mouse, Infection, Liver, Bangladesh Technical Education Board, Biotechnology, NIAID, Marketing, File, BLA, Therapy, Congress, PPMD, PUL, WMS, CMC, Distribution, International, Regenerative medicine, ID, Duchenne muscular dystrophy, Safety, Dicarboxylic acid, Biologics license application, IND, Commercialization, SARS-CoV-2, Lipid, Messenger, MicroRNA, Inflammation, FDA, Food, DMD, Priority review, Cell, COVID-19, Report, Patient, Oligonucleotide, PRV, Kidney, Tissue, Pharmaceutical industry

Further, Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement.

Key Points: 
  • Further, Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement.
  • Next steps for Cohort A: plan to report top-line data in the fourth quarter of 2024.
  • Revenues for the fourth quarter of 2023 were approximately $12.1 million compared with approximately $1.0 million for the fourth quarter of 2022.
  • Operating expenses: Total operating expenses for the fourth quarter of 2023 were approximately $13.2 million compared with approximately $9.0 million for the fourth quarter of 2022.

PPMD Celebrates 30th Anniversary and Three Decades of Progress with Annual Advocacy Conference

Retrieved on: 
월요일, 3월 4, 2024
Bangladesh Technical Education Board, NIH, FDA, Friends, Dream, Grandparent, Duchenne muscular dystrophy, Parent, Conference, Muscular dystrophy, Human, Life expectancy, Patient, Policy, Legislation, Congress, Department of Defence, Diagnosis, CDC, PPMD, Sibling, Research, Public, Pharmaceutical industry

In addition to visiting with more than 120 Congressional offices, advocates will be participating in a special PPMD-hosted town hall focused on Duchenne clinical trials to enhance the trial participation experience.

Key Points: 
  • In addition to visiting with more than 120 Congressional offices, advocates will be participating in a special PPMD-hosted town hall focused on Duchenne clinical trials to enhance the trial participation experience.
  • I am so grateful to the thousands of PPMD advocates who have made these gains possible, especially all who have traveled to our nation's capital year after year to share their stories with Congress and drive this progress," said Pat Furlong, PPMD Founding President and CEO.
  • This year's request will also include provisions to intensify the NIH's focus on cardiac health concerns of patients with Duchenne, a topic of increasing importance as people are living longer lives.
  • The clinical trials town hall session will feature patient or caregiver participants from Duchenne trials and will also include a representative from the FDA.

REGENXBIO to Host Webcast Event to Discuss New Interim Clinical Data from the Phase I/II AFFINITY DUCHENNE® Trial

Retrieved on: 
목요일, 2월 29, 2024
EST, Duchenne muscular dystrophy, Arkansas Children's Hospital, Conference, Interim, Webcast, Publication, Phase, PPMD, Episcopal conference, Pharmaceutical industry

Company to host webcast on Tuesday, March 5, 2024 at 8:30 a.m. EST

Key Points: 
  • Company to host webcast on Tuesday, March 5, 2024 at 8:30 a.m. EST
    Interim clinical data will be presented by Aravindhan Veerapandiyan, M.D., primary investigator of the AFFINITY DUCHENNE trial, at the MDA Clinical & Scientific Conference on Wednesday, March 6, 2024 at 12:00 p.m. EST
    ROCKVILLE, Md., Feb. 29, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a live webcast to discuss new interim clinical data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for the treatment of Duchenne muscular dystrophy.
  • Primary investigator Aravindhan Veerapandiyan, M.D.
  • Access: The live webcast can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com .
  • An archived replay of the webcast will be available for approximately 30 days following the presentation.

Parent Project Muscular Dystrophy Announces Pediatric Certified Duchenne Care Center, Penn State Health Children's Hospital

Retrieved on: 
수요일, 2월 21, 2024
Becker muscular dystrophy, Certification, Growth, Pediatrics, Hospital, Duchenne muscular dystrophy, History, APRN, Family, Patient, Disease, CDC, PPMD, Neurology, Research, Education, Nursing

WASHINGTON, Feb. 21, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced the expansion of their renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Penn State Health Children's Hospital in Hershey, Pennsylvania. This designation marks a significant step for the CDCC Program and continues the growth and expansion to bring comprehensive care to all people living with Duchenne and Becker muscular dystrophy.

Key Points: 
  • WASHINGTON, Feb. 21, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , announced the expansion of their renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Penn State Health Children's Hospital in Hershey, Pennsylvania.
  • Under the guidance of Dr. Ashutosh Kumar, neurologist and clinic director, Penn State Health Children's Hospital is a growing clinic that currently serves approximately 50 individuals living with Duchenne and Becker muscular dystrophy.
  • "The Penn State Health Pediatric Neuromuscular Multidisciplinary Care Team has worked tirelessly to provide a family and patient-centered approach to the care and support of children with Duchenne and Becker muscular dystrophy," said Dr. Ashutosh Kumar, pediatric neurologist and assistant professor of pediatrics and neurology at Penn State Health Children's Hospital.
  • PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne.

Parent Project Muscular Dystrophy Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program to Support Next-Gen Midi-Dystrophin Gene Therapy Development

Retrieved on: 
화요일, 2월 13, 2024
Gene, Doctor of Philosophy, Genetic disorder, Quality of life, DMD, Duchenne muscular dystrophy, Risk, Health, Protein, Patient, Knowledge, B cell, Male, PPMD, AAV, Vaccine

WASHINGTON, Feb. 13, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , is excited to announce that PPMD has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization's venture philanthropy program which provides industry funding to accelerate therapeutic development.

Key Points: 
  • WASHINGTON, Feb. 13, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , is excited to announce that PPMD has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization's venture philanthropy program which provides industry funding to accelerate therapeutic development.
  • The organization prioritizes research for next-generation and future-generation gene therapies both through PPMD's Gene Therapy Initiative and the PPMD Venture Pathways program.
  • Casey Childers, DO, PhD, Chief Executive Officer of Kinea Bio, explains:
    "We are pleased to receive this funding commitment from PPMD.
  • In addition to PPMD's significant pre-clinical and academic research funding, the organization provides funding to biopharmaceutical companies through PPMD Venture Pathways, its venture philanthropy program.