Dystrophy

Aileron Therapeutics Announces Oral Presentation of Non-Clinical Data Demonstrating ALRN-6924 Protected Human Hair Follicles and Their Stem Cells from Chemotherapy-Induced Damage at the European Society for Dermatological Research Annual Meeting 2022

Retrieved on: 
Friday, September 30, 2022
Apoptosis, Paclitaxel, Aileron, Fibroblast, Safety, Incidence, EMBO Molecular Medicine, FDA, Cyclophosphamide, Doxorubicin, ESDR, TAC, Form 10-K, Keratinocyte, Hair, Permanent Damage, Docetaxel, SID, U.S. Securities and Exchange Commission, Patient, Cancer, Breast cancer, Neutropenia, B cell, Toxic leukoencephalopathy, Coronavirus, Paus family, Private Securities Litigation Reform Act, Scalp, DNA repair, Security (finance), DNA, Taxane, Skin, Dystrophy, Annual report, Hair loss, Hair follicle, Pharmaceutical industry, Health insurance, Vaccine

It is yet another burden layered on top of their already-daunting fight against cancer, said Dr. Paus.

Key Points: 
  • It is yet another burden layered on top of their already-daunting fight against cancer, said Dr. Paus.
  • Ailerons vision is to bring chemoprotection to all patients with p53-mutated cancer regardless of the type of cancer or chemotherapy.
  • We look forward to our anticipated data readouts from that trial later this year and into 2023.
  • Aileron previously conducted in vitro studies showing that ALRN-6924 protected human fibroblasts in cell culture from multiple chemotherapies, but not p53-mutant breast cancer cells.

PTC Therapeutics' 2022 STRIVE Awards Program Funds Initiatives Supporting the Duchenne Patient Community

Retrieved on: 
Wednesday, September 7, 2022
Muscle weakness, Therapy, Muscle, PTC, Program, Degenerative disease, NASDAQ, Awareness, Time, Dystrophin, Dystrophy, Diaphragm, National Eating Disorders Association, Disease, Human, Heart, Muscular Dystrophy Association, LinkedIn, Duchenne muscular dystrophy, Diagnosis, Binary relation, Education, Organization, Student, Patient, ACDM, PTC Therapeutics, Company, Twitter, Association, Cohesion (linguistics), Knowledge, Death, Medicine, Tutoring, Pharmaceutical industry, U.K, Empowerment, Duchenne, Facebook

SOUTH PLAINFIELD, N.J., Sept. 7, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the four recipients of its 2022 STRIVE Awards for Duchenne Muscular Dystrophy. The STRIVE program provides grants to non-profit patient advocacy organizations serving the Duchenne community. This year, grants will fund projects focused on formal learning, psychological support, sexuality, and skills-based training for people with Duchenne.  

Key Points: 
  • The STRIVE program provides grants to non-profit patient advocacy organizations serving the Duchenne community.
  • "We are proud to support the 2022 grant recipients for their work in the Duchenne community through our STRIVE Awards program.
  • They have shown the broad range of needs in the Duchenne community, including programs supporting adults living with Duchenne,"said Mary Frances Harmon, senior vice president, Corporate Relations, PTC Therapeutics.
  • PTC launched the Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program in 2015 to support initiatives that benefit the Duchenne community by increasing awareness, diagnosis, and education, and fostering the development of future patient advocates.

Aileron Therapeutics Announces Late-Breaking Oral Presentation of Non-Clinical Data Demonstrating ALRN-6924 Protected Human Hair Follicles and Their Stem Cells from Chemotherapy-Induced Damage at Upcoming Society for Investigative Dermatology 2022 Annual

Retrieved on: 
Tuesday, May 10, 2022
SID, U.S. Securities and Exchange Commission, B cell, Pemetrexed, Keratinocyte, Bone marrow, Scalp, Carboplatin, Aileron, Private Securities Litigation Reform Act, Dystrophy, Breast cancer, Annual report, Topotecan, Cell cycle, Patient, Immunotherapy, DNA repair, Security (finance), Coronavirus, Hair follicle, Apoptosis, Fibroblast, Cancer, Paclitaxel, Doxorubicin, NSCLC, DNA, Form 10-K, Cyclophosphamide, Hair, Quality of life, EMBO Molecular Medicine, Docetaxel, Squamous cell carcinoma, Research, Toxic leukoencephalopathy, Risk, Vaccine, Pharmaceutical industry

These toxicities occur because chemotherapy destroys normal, healthy cells while simultaneously destroying cancer cells, said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer at Aileron.

Key Points: 
  • These toxicities occur because chemotherapy destroys normal, healthy cells while simultaneously destroying cancer cells, said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer at Aileron.
  • In addition, we found that ALRN-6924 may exert some additional benefits that could reduce the risk of long-term damage of human hair follicle stem cells by taxanes.
  • ALRN-6924 is designed to activate p53 in normal cells, which in turn upregulates p21, which pauses cell cycle in normal cells but not in p53-mutated cancer cells.
  • Aileron previously conducted in vitro studies showing that ALRN-6924 protected human fibroblasts in cell culture from multiple chemotherapies, but not p53-mutant breast cancer cells.

Jiffy Lube® and Muscular Dystrophy Association Celebrate 10th Annual Fundraising Campaign to Support the Neuromuscular Community

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Tuesday, July 27, 2021
Amyotrophic lateral sclerosis, Muscular Dystrophy Association, Neurological disorders, Organ systems, Musculoskeletal disorders, Muscular dystrophy, MDA, Jiffy Lube, Jiffy, Dystrophy, Neuromuscular disease, Muscular Dystrophy Canada

During the month of August, Jiffy Lube customers will be invited to make a donation of$3or more to MDA at participating Jiffy Lube centers.

Key Points: 
  • During the month of August, Jiffy Lube customers will be invited to make a donation of$3or more to MDA at participating Jiffy Lube centers.
  • "I'm honored to support the Muscular Dystrophy Association and Jiffy Lube's efforts to fund breakthrough science and care and commend them for their decade-long dedication of driving to do more to support MDA's mission."
  • "I'm so happy to be part of the Jiffy Lube campaign, because it is raising awareness about muscular dystrophy that I live with," said Maddee Helaire, MDA Ambassador.
  • For decades, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases.

FSHD Society offers CME-accredited masterclass on FSH muscular dystrophy

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Wednesday, July 14, 2021
Muscular dystrophy, Facioscapulohumeral muscular dystrophy, Branches of biology, Dystrophy, Medicine, Health, Genes, Max Adler, Beevor's sign

RANDOLPH, Mass., July 14, 2021 /PRNewswire-PRWeb/ --The FSHD Society is offering its first-ever CME-accredited masterclass on facioscapulohumeral muscular dystrophy (FSHD) .

Key Points: 
  • RANDOLPH, Mass., July 14, 2021 /PRNewswire-PRWeb/ --The FSHD Society is offering its first-ever CME-accredited masterclass on facioscapulohumeral muscular dystrophy (FSHD) .
  • Although FSHD is one of the most common forms of muscular dystrophy, it is often under-diagnosed, and diagnosed patients don't always benefit from the existing standards of care.
  • The FSHD Society is the world's largest research-focused patient advocacy organization for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy.
  • The FSHD Society has transformed the landscape for FSHD research and is committed to making sure that no one faces this disease alone.

Solid Biosciences Reports Inducement Grant to New Senior Vice President, Clinical Development

Retrieved on: 
Friday, July 2, 2021
Muscular dystrophy, Duchenne, Dystrophy, NASDAQ, Finance, Financial markets, Investment

CAMBRIDGE, Mass., July 02, 2021 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced the grant of an inducement award to its newly appointed Senior Vice President, Clinical Development, Roxana Dreghici.

Key Points: 
  • CAMBRIDGE, Mass., July 02, 2021 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced the grant of an inducement award to its newly appointed Senior Vice President, Clinical Development, Roxana Dreghici.
  • The inducement grant to Dr. Dreghici consisted of an option to purchase up to 230,000 shares of common stock.
  • The option has an exercise price of $3.77 per share, the closing price per share of Solid Biosciences common stock as reported by Nasdaq on July 1, 2021.
  • Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne.

Solid Biosciences Strengthens Management Team to Advance Programs for Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, July 1, 2021
Duchenne, Dystrophy, Muscular dystrophy

CAMBRIDGE, Mass., July 01, 2021 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced the hiring of Roxana Donisa Dreghici, M.D.

Key Points: 
  • CAMBRIDGE, Mass., July 01, 2021 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced the hiring of Roxana Donisa Dreghici, M.D.
  • The addition of Roxana, Caitlin and Alison brings a tremendous amount of industry experience and passion to our management team, said Ilan Ganot, Co-Founder, President and Chief Executive Officer of Solid Biosciences.
  • We continue to strengthen the leadership team at Solid Biosciences as we look to continue progressing our product pipeline and supporting the Duchenne community.
  • Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne.

Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker Muscular Dystrophy

Retrieved on: 
Saturday, June 26, 2021
Research, Hospitals, Genetics, Clinical trials, Other Health, Biotechnology, Pharmaceutical, Health, Science, Other Science, Muscular dystrophy, Branches of biology, Health, Medicine, Becker muscular dystrophy, Dystrophin, Givinostat, Dystrophy, Duchenne muscular dystrophy, Duchenne, BMD, Becker Muscular Dystrophy, Givinostat, Italfarmaco Group, BECKER MUSCULAR DYSTROPHY, GIVINOSTAT, ITALFARMACO GROUP

Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the companys proprietary histone deacetylase (HDAC) inhibitor, in 51 adult males with Becker Muscular Dystrophy (BMD).

Key Points: 
  • Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the companys proprietary histone deacetylase (HDAC) inhibitor, in 51 adult males with Becker Muscular Dystrophy (BMD).
  • The topline data was presented by Paolo Bettica on June 26, 2021, at the virtual Parent Project Muscular Dystrophy (PPMD) Annual Conference .
  • The Phase 2 trial investigating Givinostat in BMD patients was a randomized, double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03238235 ).
  • Italfarmaco is also investigating the effect of Givinostat treatment in patients with Duchenne Muscular Dystrophy (DMD) in a Phase 3 trial (ClinicalTrials.gov: NCT02851797 ).

Santhera and ReveraGen to Present Findings from Pivotal VISION-DMD Study with Vamorolone at Parent Project Muscular Dystrophy 2021 Conference

Retrieved on: 
Thursday, June 24, 2021
Glucocorticoids, Organ systems, Medicine, Medical specialties, Prodrugs, Pregnanes, Muscular dystrophy, Diols, Vamorolone, Prednisone, Dystrophy, Duchenne

The study also demonstrated superiority of both vamorolone dose levels (2 and 6mg/kg/day) versus placebo across multiple secondary endpoints.

Key Points: 
  • The study also demonstrated superiority of both vamorolone dose levels (2 and 6mg/kg/day) versus placebo across multiple secondary endpoints.
  • Vamorolone did not stunt growth, as validated in the current 24-week study, in which vamorolone 6mg/kg/day versus prednisone 0.75mg/kg/day showed a significant difference in growth velocity (p=0.02).
  • The primary endpoint of the study is TTSTAND velocity at 24 weeks comparing the 6mg/kg/day dose of vamorolone to placebo.
  • ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders.

Vita Therapeutics Raises $32 Million in Oversubscribed Series A Financing Led by Cambrian Biopharma to Advance the Development of Therapies to Treat Muscular Dystrophies

Retrieved on: 
Wednesday, June 23, 2021
Biotechnology, Other Health, Health, Genetics, Pharmaceutical, Muscular dystrophy, Dystrophy, Genetic engineering, Branches of biology, Limb-Girdle Muscular Dystrophy, Vita Therapeutics, LIMB-GIRDLE MUSCULAR DYSTROPHY, VITA THERAPEUTICS

The financing was led by Cambrian Biopharma with participation from Kiwoom Bio, SCM Life Sciences, and Early Light Ventures.

Key Points: 
  • The financing was led by Cambrian Biopharma with participation from Kiwoom Bio, SCM Life Sciences, and Early Light Ventures.
  • This oversubscribed round of financing will enable the company to take the next steps toward achieving our mission.
  • I am so glad to count Vita as a Cambrian affiliate, and I have no doubt Vita will become a genre-defining cell therapy company.
  • Vitas second therapeutic, VTA-200, is a genetically engineered iPSC derived hypoimmunogenic treatment designed to treat multiple types of muscular dystrophy.