Duchenne

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.

Satellos Bioscience Announces 2023 Q3 Financial Results and Operational Highlights

Retrieved on: 
Wednesday, November 22, 2023
Biotechnology, General Health, Pharmaceutical, Health, AAK1, Strategic management, GMP, Chemistry, Senior, MSCL, CFO, Bank statement, CMA, Regenerative medicine, Research, G&A, Therapy, MBA, Lead, Hand, MD, Michael Cross, CPA, Doctor of Philosophy, Duchenne muscular dystrophy, Travel, Pharmaceutical industry, Cryptocurrency, Duchenne, IND

Satellos Bioscience Inc. (TSXV: MSCL) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the three months ending September 30, 2023.

Key Points: 
  • Satellos Bioscience Inc. (TSXV: MSCL) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the three months ending September 30, 2023.
  • Satellos also announced that SAT-3247 would be nominated as the lead DC based on results generated by the Company during its preclinical studies.
  • Satellos had cash and cash equivalents of $44.3 million as of September 30, 2023, compared with $1.9 million at December 31, 2022.
  • Satellos’ interim condensed consolidated financial statements for the three and nine months ended September 30, 2023, and the related management’s discussion and analysis (MD&A) are available on SEDAR+ at www.sedarplus.ca .

Satellos Discloses Drug Target and Provides Development Update on Duchenne Muscular Dystrophy Program

Retrieved on: 
Tuesday, November 14, 2023
Health, Stem Cells, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, AAK1, GMP, Senior, MSCL, Dystrophin, Regeneration, IND, FAAN, Clinical trial, Lead, Duchenne muscular dystrophy, Patent, Pharmaceutical industry, Duchenne

We have generated multiple series of small molecule inhibitors of AAK1 with pharmaceutical properties designed to meet our therapeutic objectives in treating Duchenne.

Key Points: 
  • We have generated multiple series of small molecule inhibitors of AAK1 with pharmaceutical properties designed to meet our therapeutic objectives in treating Duchenne.
  • SAT-3247 also exhibited improved oral bioavailability, target specificity, and tissue distribution, when compared directly to SAT-3153.
  • “The data in our Duchenne program continue to support our drug target, mechanism of action, and proprietary small molecule leads,” said Phil Lambert, Ph.D., Chief Scientific Officer of Satellos.
  • We are excited about the improved oral bioavailability, target selectivity, and tissue distribution that SAT-3247 has exhibited in these models.

United Nations Officially Designates September 7 as World Duchenne Awareness Day

Retrieved on: 
Wednesday, November 29, 2023
World Health Organization, United, Diplomatic mission, Becker muscular dystrophy, Organization, UN, WDO, Multimedia, Permanent, Permanent representative, PPMD, Movement, WDAD, Diagnosis, Duchenne muscular dystrophy, Diplomat, United Nations, Nursing, Duchenne

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.

NS Pharma Unveils Duchenne Heroes, a Program to Inspire and Amplify the Voices of People Living with Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, November 21, 2023
Dystrophin, Patient, Caregiver, Social media, OBI Pharma, Inc., Diagnosis, Duchenne muscular dystrophy, Tourism, Duchenne

Duchenne Heroes will share their insights on WeSpeakDuchenne.com, a new educational platform for families living with Duchenne

Key Points: 
  • Duchenne Heroes will share their insights on WeSpeakDuchenne.com, a new educational platform for families living with Duchenne
    PARAMUS, N.J., Nov. 21, 2023 /PRNewswire/ -- NS Pharma, Inc. ("NS Pharma") today announced the launch of Duchenne Heroes a program created to raise awareness and champion the voices of people affected by Duchenne muscular dystrophy (Duchenne).
  • Duchenne Heroes are individuals, caregivers and family members who hope to inspire others by sharing their personal experiences in navigating diagnosis and daily life with Duchenne.
  • "The Duchenne Heroes' insights and authentic experiences can inspire families who may be navigating a Duchenne diagnosis.
  • "It means a lot to our family to participate in the Duchenne Heroes program as we know how life-changing a Duchenne diagnosis can be," said Elizabeth Cojeen, member of the Duchenne Heroes program.

Solid Biosciences Provides Third Quarter Business Update and Financial Results

Retrieved on: 
Wednesday, November 8, 2023
Research, Sale, IND, AAV, Rare disease, Acquisition, Cardiology, Solid, Caregiver, Duchenne, Choroideremia, CMO, Investment, Patient, CPVT, Hypertrophic cardiomyopathy, Pharmaceutical industry, Cryptocurrency, Pfizer

CHARLESTOWN, Mass., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the third quarter ended September 30, 2023, and provided a business update.

Key Points: 
  • “We are pleased with the continued advancement of our diversified pipeline of neuromuscular and cardiac gene therapies; including an IND submission in this quarter for SGT-003 for patients with Duchenne,” said Bo Cumbo, President and CEO of Solid Biosciences.
  • “We continue to expand our pipeline and last quarter licensed in a gene transfer program to treat catecholaminergic polymorphic ventricular tachycardia (CPVT).
  • Net loss for the third quarter of 2023 was $21.0 million, compared to $20.4 million for the third quarter of 2022.
  • Solid had $142.9 million in cash, cash equivalents, and available-for-sale securities as of September 30, 2023, compared to $213.7 million as of December 31, 2022.

Edgewise Therapeutics Reports Third Quarter 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, November 9, 2023
Biotechnology, Health, Pharmaceutical, Physical Therapy, Cardiology, LYNX, Research, FOX, DUNE, Ageing, Education, Biomarker, Muscular dystrophy, Muscular Dystrophy Association, NSAA, G&A, Myopathy, Glycogen storage disease type V, American Heart Association, WMS, R, Muscle contraction, Pharmacokinetics, Dystrophinopathy, Disorder, Glycogen, PK, Clinical trial, Duchenne muscular dystrophy, McArdle, Safety, HCM, Therapy, Patient, MESA, R9, Administration, Pharmaceutical industry, Edgewise, Duchenne, Becker

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter of 2023 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter of 2023 and recent business highlights.
  • “I’m thrilled with the progress we’ve made in 2023 across our skeletal and cardiovascular programs,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
  • Research and development (R&D) expenses were $23.8 million for the third quarter of 2023, compared to $19.6 million for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $5.7 million for the third quarter of 2023, compared to $5.8 million for the immediately preceding quarter.

Entrada Therapeutics Reports Third Quarter 2023 Financial Results

Retrieved on: 
Tuesday, November 7, 2023
FDA, United Kingdom, Clinical trial, Vertex, Food, Dystrophin, Toxicology, Neurodegenerative disease, G&A, MNGIE, Research and development, CTA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Research, Volunteering, R, Enzyme replacement therapy, Pharmacokinetics, Male, Growth, Thymidine, Entrada, Duchenne muscular dystrophy, General Administration of Sport of China, Safety, DMD, Therapy, Patient, Administration, Encephalopathy, Pharmaceutical industry, IND, Duchenne

BOSTON, Nov. 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 (EEV™)-therapeutics as a new class of medicines, today reported financial results for the third quarter ending September 30, 2023, and highlighted recent business updates.

Key Points: 
  • In July 2023, Entrada entered into a license agreement to advance the development of ENTR-501 with Pierrepont Therapeutics, Inc., a mitochondrial disease-focused company.
  • Research & Development (R&D) Expenses: R&D expenses were $22.2 million for the third quarter of 2023, compared to $19.0 million for the same period in 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $7.5 million for the third quarter of 2023, compared to $7.0 million for the same period in 2022.
  • Net Income (Loss): Net income was $35.5 million for the third quarter of 2023, compared to a net loss of $(25.1) million for the same period in 2022.

Parent Project Muscular Dystrophy Provides $250,000 in Funding to Rare Disease Research, LLC to Establish New Clinical Trial Site

Retrieved on: 
Tuesday, November 7, 2023
Principal, Office of Rare Diseases Research, Neurology, MD, Doctor of Philosophy, Pediatrics, Drug development, RDR, PPMD, Disease, Duchenne muscular dystrophy, Travel, The Duke's Children, Patient, Pharmaceutical industry, Research, Duchenne

WASHINGTON, Nov. 7, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , today announced that PPMD has provided $250,000 in funding to Rare Disease Research, LLC (RDR), to open a second independent rare disease clinical research site in the Raleigh-Durham metropolitan area in North Carolina.

Key Points: 
  • WASHINGTON, Nov. 7, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , today announced that PPMD has provided $250,000 in funding to Rare Disease Research, LLC (RDR), to open a second independent rare disease clinical research site in the Raleigh-Durham metropolitan area in North Carolina.
  • With this funding commitment, PPMD aims to support RDR's creation of an avenue that increases patient access to clinical trials while expediting the advancement of treatments for Duchenne.
  • RDR has strategically appointed Edward Smith, MD, a distinguished pediatric neurologist renowned for his expertise in neuromuscular clinical trials, as the principal investigator and clinical director of the new trial site in Hillsborough, NC.
  • Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation, emphasizes the organization's innovative approach in advancing Duchenne treatments.

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Sarepta Therapeutics, Inc. - SRPT

Retrieved on: 
Sunday, November 5, 2023
Sarepta, FDA, OTP, Food, Prior, May 29, News, SRPT, Pomerantz, LLP, Duchenne, Duchenne muscular dystrophy, DMD, BLA, Pharmaceutical industry, Cryptocurrency, Therapy

NEW YORK, Nov. 05, 2023 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Sarepta Therapeutics, Inc. (“Sarepta” or the “Company”) (NASDAQ: SRPT).

Key Points: 
  • NEW YORK, Nov. 05, 2023 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Sarepta Therapeutics, Inc. (“Sarepta” or the “Company”) (NASDAQ: SRPT).
  • Such investors are advised to contact Robert S. Willoughby at [email protected] or 888-476-6529, ext.
  • The investigation concerns whether Sarepta and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.