Becker muscular dystrophy

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

Retrieved on: 
Friday, March 22, 2024
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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.

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

Retrieved on: 
Wednesday, February 21, 2024
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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.

Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Duchenne Muscular Dystrophy (Duchenne)

Retrieved on: 
Tuesday, February 13, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Becker muscular dystrophy, Fast Track, Therapy, LYNX, FOX, Food, ODD, Survival, Ageing, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of Duchenne.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of Duchenne.
  • EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker muscular dystrophy (Becker).
  • The FDA previously granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne and Becker, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designation for the treatment of Becker.
  • “Every day is important for individuals living with Duchenne and their families, and we are pleased that the FDA granted Fast Track designation,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.

Edgewise Therapeutics Announces Pricing of $240 Million Underwritten Offering of Common Stock

Retrieved on: 
Friday, January 19, 2024
Biotechnology, Pharmaceutical, Health, Cardiology, Janus, Citadel, Myopathy, Patient, Therapy, Research, SVB Securities, OrbiMed, Edgewise, Prospectus, Novo Holdings A/S, Form, Sale, Becker muscular dystrophy, ATTN:, Montgomery Street, Attention, Telephone, State Street

Edgewise Therapeutics, Inc. (NASDAQ: EWTX), a leading muscle disease biopharmaceutical company, today announced the pricing of an underwritten offering of 21,818,182 shares of its common stock at an offering price of $11.00 per share.

Key Points: 
  • Edgewise Therapeutics, Inc. (NASDAQ: EWTX), a leading muscle disease biopharmaceutical company, today announced the pricing of an underwritten offering of 21,818,182 shares of its common stock at an offering price of $11.00 per share.
  • Edgewise anticipates gross proceeds from the offering to be approximately $240 million, before deducting underwriting discounts and commissions and offering expenses.
  • The closing of the offering is expected to occur on January 23, 2024, subject to the satisfaction of customary closing conditions.
  • A prospectus supplement and accompanying prospectus relating to the offering will also be filed with the SEC.

CureDuchenne Announces Educational Events for Families and Caregivers of Individuals with Duchenne or Becker Muscular Dystrophy

Retrieved on: 
Tuesday, January 23, 2024
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NEWPORT BEACH, Calif., Jan. 23, 2024 /PRNewswire/ -- CureDuchenne, a global leader in research, patient care and innovation for improving and extending the lives of those living with Duchenne muscular dystrophy, has announced its schedule of CureDuchenne Cares workshops and dinner sessions for the first half of the year, as well as its annual conference. CureDuchenne Cares provides interactive outreach programs to educate, empower, support and improve connections among families and caregivers of individuals with Duchenne muscular dystrophy, one of the most common and severe forms of muscular dystrophy, or Becker muscular dystrophy.

Key Points: 
  • CureDuchenne Cares provides interactive outreach programs to educate, empower, support and improve connections among families and caregivers of individuals with Duchenne muscular dystrophy, one of the most common and severe forms of muscular dystrophy, or Becker muscular dystrophy.
  • "At CureDuchenne, we are committed to accelerating a cure for Duchenne muscular dystrophy and fostering a community where families and caregivers can find strength, support and hope," said Debra Miller, founder and CEO of CureDuchenne.
  • "Our CureDuchenne Cares events are designed to be more than just educational; they are a lifeline, connecting individuals to a network of care, understanding, and the latest advancements in treatment.
  • We believe in empowering our community with knowledge and resources, ensuring that no one faces the challenges of Duchenne or Becker alone."

Edgewise Therapeutics Launches First of Its Kind Educational Website Dedicated to the Becker Muscular Dystrophy Community

Retrieved on: 
Monday, December 18, 2023
Health, Technology, Other Health, Internet, Pharmaceutical, Biotechnology, Human, Becker muscular dystrophy, Organization, Therapy, Cardiomyopathy, Disease, Disorder, Genetic disorder, Heart failure, Weakness, Caregiver, Diagnosis, Facial muscles, Cardiology, Heart transplantation, HIV disease progression rates, Nursing, Pharmaceutical industry, Becker

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the launch of an educational website dedicated to the Becker muscular dystrophy (Becker) community: www.beckermusculardystrophy.com .

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the launch of an educational website dedicated to the Becker muscular dystrophy (Becker) community: www.beckermusculardystrophy.com .
  • Becker is a genetic disorder, mostly impacting males, with an estimated 5,000 individuals living with the disease in the United States.
  • Individuals with Becker experience progressive muscle degeneration and weakness due to contraction-induced muscle damage, leading to the eventual loss of muscle function.
  • In developing the website, Edgewise incorporated input from the Becker community and patient advocacy organizations.

Edgewise Receives Orphan Drug and Rare Pediatric Disease Designations for Its Muscular Dystrophy Program from FDA

Retrieved on: 
Thursday, November 30, 2023
Research, Neurology, FDA, Genetics, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Becker muscular dystrophy, Therapy, ODD, Dystrophinopathy, Marketing, Duchenne muscular dystrophy, PDUFA, Fast Track, Pharmaceutical industry, Duchenne, Becker

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne.
  • EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker.
  • The FDA previously granted Fast Track designation for the investigation and development of EDG-5506 for the treatment of Becker.
  • “Receiving orphan drug and rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating individuals with Duchenne and Becker,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.

United Nations Officially Designates September 7 as World Duchenne Awareness Day

Retrieved on: 
Wednesday, November 29, 2023
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NEW YORK, Nov. 29, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) and the World Duchenne Organization (WDO), along with WDO's members and affiliates, proudly herald the United Nations marking a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day (WDAD) , set to be observed annually from 2024.

Key Points: 
  • NEW YORK, Nov. 29, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) and the World Duchenne Organization (WDO), along with WDO's members and affiliates, proudly herald the United Nations marking a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day (WDAD) , set to be observed annually from 2024.
  • This adoption of the resolution "World Duchenne Awareness Day" is the UN's first formal acknowledgment of a day dedicated to a rare disease.
  • The success of this initiative can be attributed to the collective efforts of individuals and Duchenne-focused organizations around the world.
  • As we celebrate this momentous occasion, the World Duchenne Organization and its member organizations eagerly anticipate using this recognition to further educate and engage the global community in combating Duchenne muscular dystrophy.

PPMD Applauds FDA for Approval of AGAMREE® (Vamorolone) for Duchenne Patients Ages Two Years and Older

Retrieved on: 
Friday, October 27, 2023
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AGAMREE® marks a significant milestone as the second therapy approved for Duchenne that is applicable regardless of the specific genetic mutation.

Key Points: 
  • AGAMREE® marks a significant milestone as the second therapy approved for Duchenne that is applicable regardless of the specific genetic mutation.
  • Today's approval has the potential to offer patients a therapy option with better tolerability, addressing a significant unmet need in our community," said PPMD Founding President and CEO Pat Furlong.
  • We also want to commend FDA leadership for recognizing the potential of AGAMREE® to make a difference in the lives of individuals living with Duchenne," Furlong added.
  • "PPMD is pleased to have collaborated with other foundations and community partners to support the early development of vamorolone.

Duchenne Muscular Dystrophy Added to New York State's Newborn Screening Panel

Retrieved on: 
Wednesday, October 25, 2023
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WASHINGTON, Oct. 25, 2023 /PRNewswire/ -- In a monumental stride towards improving the lives of children affected by Duchenne muscular dystrophy (Duchenne), Parent Project Muscular Dystrophy (PPMD) proudly announces that Governor Hochul of New York State (NYS) has signed bill S6814/A5042, making Duchenne newborn screening mandatory for all babies born in the state. This remarkable milestone comes after years of dedicated advocacy by PPMD, in collaboration with partners and families, to break down barriers to treatment and care for the Duchenne community.

Key Points: 
  • Another Duchenne Newborn Screening win—every baby born in New York State to be screened for Duchenne muscular dystrophy!
  • WASHINGTON, Oct. 25, 2023 /PRNewswire/ -- In a monumental stride towards improving the lives of children affected by Duchenne muscular dystrophy (Duchenne), Parent Project Muscular Dystrophy (PPMD) proudly announces that Governor Hochul of New York State (NYS) has signed bill S6814/A5042, making Duchenne newborn screening mandatory for all babies born in the state.
  • New York State has paved the path in advancing Duchenne newborn screening, notably through a pilot program carried out from 2019 to 2021.
  • New York State is now the second state, after Ohio, to mandate newborn screening for Duchenne.