Duchenne

Entrada Therapeutics Awards Funding to Non-Profit Organizations Advancing Diversity, Equity, Inclusion and Accessibility within the Duchenne Muscular Dystrophy Community

Retrieved on: 
Thursday, September 7, 2023
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BOSTON, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEVÔ) therapeutics as a new class of medicines, today announced recipients of its inaugural Entrada Diversity, Representation, Equity and Advocacy MatterS (DREAMS) Grant Program. Entrada DREAMS grant recipients – The Akari Foundation, Neurology and Neuromuscular Care Center and Parent Project Muscular Dystrophy (PPMD) – will each be awarded $25,000 to support initiatives that aim to better identify, understand and reach those within the Duchenne community who are currently underrepresented or underserved.

Key Points: 
  • Funding provided by Entrada will support the clinic in hiring its first mental health professional.
  • This individual will help to address gaps in socioeconomic health needs by providing emotional and social support to people and families living with Duchenne.
  • Parent Project Muscular Dystrophy (PPMD) Foundation is a patient advocacy organization fighting to end Duchenne by helping those living with the condition to lead longer, stronger lives.
  • On behalf of the DREAMS Review Committee, we congratulate these three vital organizations and we look forward to seeing the lasting impact that their work will continue to have on the Duchenne community.”

Santhera Announces Half-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, September 7, 2023
Vamorolone, Marketing, European Medicines Agency, Lead, Prescription Drug User Fee Act, Committee, U.S. FDA, Patient, Committee for Medicinal Products for Human Use, News, Duchenne muscular dystrophy, Glucocorticoid, Becker muscular dystrophy, European Commission, EMA, CEST, PDUFA, Rare, FDA, Safety, Health care, LR, Duchenne, Publication, MAA, MHRA, CHMP, Catalyst Pharmaceuticals, Partnership, European Directive on Traditional Herbal Medicinal Products, NDA, BMD, EC, BST, Medical device, Pharmaceutical industry, Security (finance), Idebenone, EU, Chiesi Farmaceutici, LHON, Atlantic Ocean, Catalysis, DMD

Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.

Key Points: 
  • Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.
  • Total consideration to Santhera is up to USD 231 million (including equity investment) plus royalty payments from product sales.
  • After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment).
  • During the six months ending June 30, 2023, Santhera committed to the sale of Raxone/idebenone upon settlement of the French case and the transaction closed on July 28, 2023.

Sarepta Therapeutics Announces Recipients of the 6th Annual Route 79, The Duchenne Scholarship Program, for the 2023-2024 Academic Year

Retrieved on: 
Thursday, September 7, 2023
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced 25 recipients of Route 79, The Duchenne Scholarship Program for the 2023-2024 academic year.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced 25 recipients of Route 79, The Duchenne Scholarship Program for the 2023-2024 academic year.
  • Of the 25 recipients, academic scholarships will be awarded to 20 individuals living with Duchenne muscular dystrophy and to five siblings of individuals living with Duchenne.
  • “On behalf of Sarepta and the selection committee, we are proud to announce the recipients of Route 79, The Duchenne Scholarship Program, for the 2023-2024 academic year.
  • Recipients of the scholarship are chosen by an independent selection committee composed of Duchenne community members, who consider each applicant’s community involvement and personal essay.

PTC Therapeutics' 2023 STRIVE Awards Program Funds Initiatives Supporting the Duchenne Patient Community

Retrieved on: 
Thursday, September 7, 2023
PTC Therapeutics, CSE, University, Disability, PTC, Quality of life, Social, University of Queensland, Duchenne muscular dystrophy, Wheelchair, Organization, NASW, MLA, Patient, Transition, Blue Angels, Sport, Health, Therapy, Disease, Pharmaceutical industry, Nursing, Duchenne

SOUTH PLAINFIELD, N.J., Sept. 7, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the two recipients of its 2023 STRIVE Awards for Duchenne Muscular Dystrophy. Through the STRIVE program, the company awards grants to non-profit patient advocacy organizations serving the Duchenne community. This year's grants will fund projects focused on developing a digital health app to support patients transitioning to adulthood in Australia and the expansion of a power soccer (also known as powerchair football) program in Brazil.

Key Points: 
  • SOUTH PLAINFIELD, N.J., Sept. 7, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the two recipients of its 2023 STRIVE Awards for Duchenne Muscular Dystrophy.
  • Through the STRIVE program, the company awards grants to non-profit patient advocacy organizations serving the Duchenne community.
  • "As we near a decade of the STRIVE Awards program, we are proud to continue to provide organizations with support to expand their services for the Duchenne community."
  • "It has been such a rewarding experience to be part of the judging panel for the STRIVE Awards Program.

PepGen Inc. Presents PGN-EDODM1 Preclinical Data Supporting the Company’s Enhanced Delivery Oligonucleotide Platform and PGN-EDODM1 Program at Two Medical Conferences

Retrieved on: 
Wednesday, September 6, 2023
Neuromuscular disease, PGN, Heart, Human, Myotonic Dystrophy Foundation, Duchenne muscular dystrophy, Therapy, NMD, DM1, Safety, Peptide, Cell, RNA, Duchenne, Myotonic dystrophy, Myotonia, EDO, DMD, Electrophysiology, NHP, Conference, MDF, Oligonucleotide, Potential, Disease, Pharmaceutical industry, Fine chemical, Tolerability, Pharmacokinetics, Biology

BOSTON, Sept. 06, 2023 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced key highlights from the upcoming presentation of preclinical non-human primate (NHP) data supporting PepGen’s proprietary Enhanced Delivery Oligonucleotide (EDO) platform at the 6th Ottawa International Conference on Neuromuscular Disease and Biology (NMD) being held on September 7-9, 2023 in Ottawa, ON, Canada, and at the 2023 Myotonic Dystrophy Foundation (MDF) Annual Conference being held on September 7-9, 2023 in Washington, D.C.

Key Points: 
  • Additionally, the presentation reported that EDO technology was also shown to improve in vivo nuclear delivery, with 72% of skeletal muscle nuclei in NHPs positive for oligonucleotide following two doses at 30mg/kg.
  • PepGen previously demonstrated that a single dose of 30 mg/kg of PGN-EDODM1 corrected myotonia in the DM1 HSALR mouse model as measured by electrophysiology.
  • Treatment emergent adverse events in this study at these dose levels were transient, mild (grade 1), and reversible and did not require intervention.
  • “We have previously demonstrated the ability of the EDO technology to deliver up to 50 nM of oligonucleotide to biceps in humans following a single dose.

FibroGen Announces Topline Results from LELANTOS-2, a Phase 3 Clinical Study of Pamrevlumab in Ambulatory Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, August 29, 2023
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SAN FRANCISCO, Aug. 29, 2023 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced topline data from the Phase 3 LELANTOS-2 trial of pamrevlumab for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) on background systemic corticosteroids. The study did not meet the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) total score from baseline to week 52 (placebo-corrected mean difference -0.528 points; 95% CI -2.308 to 1.251; p=0.5553). Secondary endpoints measured by change from baseline at week 52 in 4-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test were also not met.

Key Points: 
  • SAN FRANCISCO, Aug. 29, 2023 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced topline data from the Phase 3 LELANTOS-2 trial of pamrevlumab for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) on background systemic corticosteroids.
  • “We are deeply disappointed that the LELANTOS-2 study did not meet its primary endpoint,” said Thane Wettig, Interim Chief Executive Officer, FibroGen.
  • “We are grateful for the courageous efforts of patients, their caregivers, the advocacy community, and the trial investigators who have contributed to this important clinical study.
  • The Company plans to communicate the full results of the LELANTOS-2 study at an upcoming medical forum.

Parent Project Muscular Dystrophy Joins Forces with Others on World Duchenne Awareness Day, Announces Duchenne Action Month this September

Retrieved on: 
Monday, August 28, 2023
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WASHINGTON, Aug. 28, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will join forces with fellow Duchenne-specific organizations from around the world to encourage families affected by Duchenne to share their story for World Duchenne Awareness Day on September 7. An initiative led by the World Duchenne Organization, World Duchenne Awareness Day aims to raise awareness about Duchenne and Becker muscular dystrophy.

Key Points: 
  • WASHINGTON, Aug. 28, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will join forces with fellow Duchenne-specific organizations from around the world to encourage families affected by Duchenne to share their story for World Duchenne Awareness Day on September 7.
  • An initiative led by the World Duchenne Organization, World Duchenne Awareness Day aims to raise awareness about Duchenne and Becker muscular dystrophy.
  • This year's theme for World Duchenne Awareness Day is "Duchenne: Breaking Barriers."
  • If you would like to learn more about World Duchenne Awareness Day or Duchenne Action Month, including details on how you can participate, please visit the PPMD website .

New Gene Therapy Gives Hope to Boy with Duchenne Muscular Dystrophy

Retrieved on: 
Friday, August 25, 2023
Exercise, Optimism, Duchenne muscular dystrophy, Gene, Parent, Atrophy, Hope, B cell, DSM-IV codes, Research, Temperature, Pharmacy, CHKD, SpongeBob SquarePants, Grief, DMD, Patient, Child, Observation, FDA, Therapy, Family, Muscular dystrophy, Disease, Dairy, Nursing, Medical device, Duchenne

Karson was at CHKD to receive a newly approved gene therapy for Duchenne muscular dystrophy, which he was diagnosed with at 20 months of age.

Key Points: 
  • Karson was at CHKD to receive a newly approved gene therapy for Duchenne muscular dystrophy, which he was diagnosed with at 20 months of age.
  • Duchenne is a devastating genetic illness that causes progressive muscular wasting.
  • Proud, who specializes in neuromuscular conditions, has only been able to treat the symptoms of Duchenne, mainly with oral corticosteroids.
  • “Elevidys is a gene therapy that spurs the development of a protein similar to the protein missing in the muscle cells of children with Duchenne,” she says.

Muscular Dystrophy Association International Association of Fire Fighters Launch 420 Fill the Boot Fundraisers in Cities Across America Labor Day Weekend for the Muscular Dystrophy Association

Retrieved on: 
Monday, August 21, 2023
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"All of us at IAFF have remained committed to the Muscular Dystrophy Association as our charity of choice since 1954.

Key Points: 
  • "All of us at IAFF have remained committed to the Muscular Dystrophy Association as our charity of choice since 1954.
  • While we Fill the Boot year-round, Labor Day Weekend is a special time where fire fighters across the country continue to collect critical funds in the community – one dollar at a time.
  • It’s the start of National Muscular Dystrophy Awareness Month and is an important time of year for this longstanding tradition.
  • Recently, the first-ever FDA approved gene therapy for Duchenne muscular dystrophy became a reality.

Solid Biosciences Provides Second Quarter Business Update and Financial Results

Retrieved on: 
Monday, August 14, 2023
Insurance, Duchenne muscular dystrophy, Ataxia, Solid, Research, Duchenne, Sale, BAG3, Investigational New Drug, Patient, Investment, IND, Pharmaceutical industry, Video game, Birth control

CHARLESTOWN, Mass., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the second quarter ended June 30, 2023, and provided a business update.

Key Points: 
  • “We made strong progress this quarter across multiple programs in our pipeline of genetic medicines and remain on track to submit an IND for SGT-003 in the fourth quarter of this year,” said Bo Cumbo, President and CEO of Solid Biosciences.
  • Solid also continues to advance preclinical programs in both cardiac and neuromuscular indications as well as platform enabling capsids.
  • There were no collaboration revenues for the second quarter of 2023, compared to $6.2 million, for the second quarter of 2022.
  • Net loss for the second quarter of 2023 was $24.6 million, compared to $25.1 million for the second quarter of 2022.