Duchenne

Sarepta Therapeutics Announces Third Quarter 2023 Financial Results and Recent Corporate Developments

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Wednesday, November 1, 2023
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the third quarter 2023.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the third quarter 2023.
  • Total net product revenue stands at $309.3 million, growing 49 percent over the same quarter last year.
  • These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP.
  • All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy

Retrieved on: 
Monday, October 30, 2023
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Full results from EMBARK will be shared at future medical meetings and publication will be pursued in a medical journal.

Key Points: 
  • Full results from EMBARK will be shared at future medical meetings and publication will be pursued in a medical journal.
  • “The consistency of the positive effect across all timed function tests and age groups provides evidence of a meaningful treatment effect.
  • On October 30, 2023, at 4:30 p.m. Eastern time, Sarepta will host a conference call and webcast to discuss these results.
  • Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing.

Defeat Duchenne Canada and Parent Project Muscular Dystrophy Award $300,000 (USD) Clinical Fellowship in Duchenne Endocrinology and Bone Fragility

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Friday, November 3, 2023
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WASHINGTON and OTTAWA, ON, Nov. 3, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), and Defeat Duchenne Canada, Canada's leading charity fighting to defeat Duchenne, today announced a collaborative research award of $300,000 (USD) in support of two Clinical Fellowships in Duchenne Endocrinology and Bone Fragility. The three-year award will sponsor the extended fellowship of Dr. Kim Phung, as well as new fellow Dr. Rana Halloun, under the guidance of Dr. Leanne Ward, Professor of Pediatrics and Research Chair in Pediatric Bone Disorders at the University of Ottawa.

Key Points: 
  • Duchenne is the most common muscular dystrophy in children, affecting approximately one in 5,000 live male births.
  • Duchenne is caused by a change in the dystrophin gene that leads to progressive muscle weakness.
  • In 2021, Dr. Phung was awarded a two-year fellowship in pediatric endocrine and bone diseases at the University of Ottawa, with specific focus on endocrine and bone complications of Duchenne.
  • Ward and Phung will delve into the latest developments and crucial topics related to bone health, growth, puberty, and adrenal insufficiency for Duchenne muscular dystrophy.

Nicklaus Children's Hospital Now Administering the First FDA-Approved Gene Therapy for Duchenne Muscular Dystrophy

Retrieved on: 
Wednesday, November 1, 2023
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MIAMI, Nov. 1, 2023 /PRNewswire/ -- Nicklaus Children's Hospital announced it has treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy.

Key Points: 
  • MIAMI, Nov. 1, 2023 /PRNewswire/ -- Nicklaus Children's Hospital announced it has treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy.
  • Nicklaus Children's is one of the first in the nation and one of only three hospitals in Florida offering this new treatment.
  • Jacob, who just turned 6 and resides in Miami's Little Havana neighborhood was treated on October 12.
  • Our use of emerging treatments is one more reason why Nicklaus Children's is the hospital where children matter most."

Santhera Receives U.S. FDA Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Friday, October 27, 2023
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“We are delighted to secure FDA approval which comes just weeks after the positive opinion from the CHMP of the European Medicines Agency.

Key Points: 
  • “We are delighted to secure FDA approval which comes just weeks after the positive opinion from the CHMP of the European Medicines Agency.
  • This is a hugely important moment for DMD patients who need an efficacious and well-tolerated therapy for this debilitating condition,” said Dario Eklund, CEO of Santhera.
  • “Steroids are considered standard of care for Duchenne, due to their valuable role in slowing disease progression.
  • Subject to approval, AGAMREE will be the only medicinal product fully approved in both territories, the EU and the U.S., for the treatment of DMD.

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.

Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA

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Thursday, October 26, 2023
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New York, Oct. 26, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE® (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD).

Key Points: 
  • New York, Oct. 26, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE® (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD).
  • This multi-functional drug shows potent inhibition of pro-inflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor, high-affinity antagonism for the mineralocorticoid receptor, and membrane stabilization properties.
  • This novel therapy, supported in part by MDA Venture Philanthropy, demonstrates similar efficacy to traditional corticosteroids with reduced negative downstream impacts or side effects.
  • “My son was diagnosed with Duchenne muscular dystrophy, and he was the first person dosed with vamorolone right before his 7th birthday.

Edgewise Therapeutics Announces Expansion of their EDG-5506 Clinical Program in Duchenne Muscular Dystrophy (Duchenne)

Retrieved on: 
Thursday, October 26, 2023
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Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced an expansion of their clinical development program of EDG-5506, an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced an expansion of their clinical development program of EDG-5506, an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne.
  • The Company is initiating FOX, a new Phase 2 placebo-controlled trial in children and adolescent boys with Duchenne who have been previously treated with gene therapy.
  • The FOX trial will assess the effect of EDG-5506 over 12 weeks on safety, pharmacokinetics (PK) and biomarkers of muscle damage.
  • The study will also explore changes in functional measures, such as the North Star Ambulatory Assessment (NSAA) and self-reported/caregiver-reported outcomes.

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.

Lisa McCoy Joins Defeat Duchenne Canada as New CEO

Retrieved on: 
Tuesday, October 24, 2023
Heart, American Cancer Society, Jesse's Journey, Heart and Stroke Foundation of Canada, Tourette Canada, Multiple Sclerosis Society, Duchenne muscular dystrophy, Executive, Stroke, Patient, Canadian Cancer Society, Management, Nursing, Duchenne

Defeat Duchenne Canada appoints Lisa McCoy as new CEO.

Key Points: 
  • Defeat Duchenne Canada appoints Lisa McCoy as new CEO.
  • "I am thrilled to join the Defeat Duchenne Canada team and contribute to the vital vision: a future without Duchenne," said Lisa McCoy.
  • "We are confident that Lisa will provide the visionary leadership required to take Defeat Duchenne Canada to new heights," said Brain Atkinson, Chair of the Board of Directors at Defeat Duchenne Canada.
  • Lisa McCoy assumes her role as CEO of Defeat Duchenne Canada immediately.