Becker muscular dystrophy

Edgewise Receives European Medicines Agency (EMA) Orphan Drug Designations for Sevasemten (EDG-5506) for the Treatment of Becker and Duchenne Muscular Dystrophies

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Tuesday, April 23, 2024

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne).

Key Points:

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne).
Sevasemten is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage.
Sevasemten is currently in late-stage clinical trials for individuals with Becker and is also being studied in Duchenne.
Sevasemten has previously achieved notable milestones by securing U.S. Food and Drug Administration (FDA) Orphan Drug Designation for the treatment of Becker and Duchenne, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designations for the treatment of Becker and Duchenne.

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

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Wednesday, April 17, 2024

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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

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Monday, April 15, 2024

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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 .

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

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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

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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.

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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.
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)

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Tuesday, February 13, 2024

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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

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Friday, January 19, 2024

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.

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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

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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

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Monday, December 18, 2023

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

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Thursday, November 30, 2023

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.