Bone marrow failure

Advisory - Certain lots of Tinactin aerosol sprays for athlete's foot recalled

Retrieved on: 
Tuesday, October 5, 2021
Dizziness, Leukemia, Cancer, Atmosphere, Headache, Bone marrow failure, Inhalation, Anemia, Benzene, Life, Health Canada, Tolnaftate, Canada, Foot, Skin, Medical device, Oil

Product:Tinactin Aerosol Powder, Tinactin Chill Deodorant Powder Spray, and Tinactin Chill Liquid Spray

Key Points: 
  • Product:Tinactin Aerosol Powder, Tinactin Chill Deodorant Powder Spray, and Tinactin Chill Liquid Spray
    Issue:Bayer Inc. is recalling certain lots due to elevated levels of benzene, which may pose serious health risks.
  • Bayer Inc. is recalling six lots (lot numbers beginning with TN) of Tinactin Aerosol Powder, Tinactin Chill Deodorant Powder Spray, and Tinactin Chill Liquid Spray due to elevated levels of benzene.
  • Frequent and long-term exposure (e.g., through the skin and by inhalation) to elevated levels of benzene may pose serious health risks.
  • These sprays are antifungal products used for the treatment and prevention of athlete's foot.

Ready to Treat Over 80 Life-Threatening Diseases, Discover the Potential of Cord Blood during World Cord Blood Day 2021

Retrieved on: 
Tuesday, October 5, 2021
Blood, Social media, Temperature, COVID-19, Regenerative medicine, Cord blood bank, Umbilical cord, Leukemia, Parent, Accreditation, World Marrow Donor Association, Bone marrow, Stroke, Partner, Student, Bone marrow failure, Hospital, Nursing, Method, Lymphoma, Cerebral palsy, Education, FACT, Child, Placenta, Sickle cell disease, Research, Waste, Thalassemia, Foundation, Laboratory, NMDP, Autism, Birth control, Pharmaceutical industry

Cord blood is the blood left in the umbilical cord and placenta following the birth of a child.

Key Points: 
  • Cord blood is the blood left in the umbilical cord and placenta following the birth of a child.
  • During World Cord Blood Day 2021, participants will learn how cord blood is used to treat over 80 life-threatening diseases such as leukemia and lymphoma, bone marrow failure, immune deficiency diseases and inherited blood disorders such as thalassemia and sickle cell disease.
  • Organized by Save the Cord Foundation, a 501c3 non-profit, World Cord Blood Day 2021 is officially sponsored by QuickSTAT Global Life Science Logistics, recognized leader in medical shipping and healthcare logistics.
  • "QuickSTAT, part of Kuehne+Nagel, is proud to sponsor the 5th annual World Cord Blood Day to help support and educate the healthcare community and expectant parents about the life-saving value of cord blood stem cells.

Rocket Pharmaceuticals to Participate in the Chardan Virtual 5th Annual Genetic Medicines Conference

Retrieved on: 
Thursday, September 30, 2021
Other Health, Health, Genetics, Pharmaceutical, Clinical Trials, Heart failure, PKD, FA, Target, Conference, NASDAQ, Webcast, Patient, Bone marrow failure, Medication, Danon disease, Lists of diseases, Pyruvate kinase deficiency, LVV, Genetic disorder, Heart, Osteopetrosis, AAV, IMO, Fanconi anemia, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announced that Gaurav Shah, M.D., chief executive officer, will participate in a fireside chat on Tuesday, October 5, 2021, at 10:30 am ET at the Chardan Virtual 5th Annual Genetic Medicines Conference.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announced that Gaurav Shah, M.D., chief executive officer, will participate in a fireside chat on Tuesday, October 5, 2021, at 10:30 am ET at the Chardan Virtual 5th Annual Genetic Medicines Conference.
  • The live webcast of the fireside chat will be accessible via Rockets website on the Events page.
  • An archived copy of the webcast will be available on the Rocket website for 30 days after the event.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.

Secura Bio Announces European Distribution Agreement for COPIKTRA® with Clinigen Healthcare Limited

Retrieved on: 
Wednesday, September 22, 2021
Follicular lymphoma, Cancer, Infection, European Medicines Agency, PI3K, AIM, FDA, Patient, PTCL, Bone marrow failure, Physician, Adult, Diarrhea, Anemia, CEO, Group, United Kingdom, Growth, CLL, Asia, Maintenance, Lymphatic system, Survival, Quality of life, CLIN, Colitis, Peripheral T-cell lymphoma, Spruce Production Division, Enzyme, Chronic lymphocytic leukemia, Pharmaceutical industry

Secura Bio will promote and distribute COPIKTRA directly in Germany and the United Kingdom.

Key Points: 
  • Secura Bio will promote and distribute COPIKTRA directly in Germany and the United Kingdom.
  • Secura Bio is currently working with the haematology community and relevant authorities to ensure COPIKTRA is widely reimbursed and commercially launched across Europe in the coming months.
  • We are excited to be working with Clinigen to ensure successful market access, commercialization and supply of Copiktra across the European geographies".
  • Sam Herbert, Chief Operating Officer and Head of Products Division at Clinigen said that"Clinigen is very pleased to be partnering with Secura Bio.

Secura Bio Announces European Distribution Agreement for COPIKTRA® with Clinigen Healthcare Limited

Retrieved on: 
Wednesday, September 22, 2021
Follicular lymphoma, Cancer, Infection, European Medicines Agency, PI3K, AIM, FDA, Patient, PTCL, Bone marrow failure, Physician, Adult, Diarrhea, Anemia, CEO, Group, United Kingdom, Growth, CLL, Asia, Maintenance, Lymphatic system, Survival, Quality of life, CLIN, Colitis, Peripheral T-cell lymphoma, Spruce Production Division, Enzyme, Chronic lymphocytic leukemia, Pharmaceutical industry

Secura Bio will promote and distribute COPIKTRA directly in Germany and the United Kingdom.

Key Points: 
  • Secura Bio will promote and distribute COPIKTRA directly in Germany and the United Kingdom.
  • Secura Bio is currently working with the haematology community and relevant authorities to ensure COPIKTRA is widely reimbursed and commercially launched across Europe in the coming months.
  • We are excited to be working with Clinigen to ensure successful market access, commercialization and supply of Copiktra across the European geographies".
  • Sam Herbert, Chief Operating Officer and Head of Products Division at Clinigen said that"Clinigen is very pleased to be partnering with Secura Bio.

Rocket Pharmaceuticals Presents Clinical Data from RP-A501 Trial in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021

Retrieved on: 
Tuesday, September 14, 2021
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Autophagosome, Medication, IMO, Tissue, Genetic disorder, Cardiac muscle, Patient, Immunohistochemistry, Vacuole, Marketing, LVV, Private Securities Litigation Reform Act, Autophagy, Lists of diseases, Glycogen, Danon disease, Fanconi anemia, Safety, BNP, NASDAQ, Osteopetrosis, COVID-19, Heart Failure Society of America, Bone marrow failure, Heart failure, Lifting, Male, FA, PKD, Risk, AAV, SEC, Pyruvate kinase deficiency, Prevalence, Pharmaceutical industry, Vaccine, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, ROCKET PHARMACEUTICALS, INC.

Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.

Key Points: 
  • Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
  • In these two patients, a substantial improvement of a key marker of heart failure, B-type natriuretic peptide (BNP), was also observed.
  • RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Announces Upcoming Danon Disease Presentation at the Heart Failure Society of America Annual Scientific Meeting 2021

Retrieved on: 
Friday, September 10, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021.
  • The meeting will take place September 10-13, 2021 at the Gaylord Rockies Hotel and Conference Center in Aurora, Colorado.
  • Details for Rockets oral presentation are as follows:
    RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Appoints Isabel Carmona, J.D., as Chief Human Resources Officer

Retrieved on: 
Tuesday, September 7, 2021
Professional Services, Health, Genetics, Human Resources, Pharmaceutical, Biotechnology, Rocket Pharmaceuticals Leadership, Rocket Pharmaceuticals, Inc., ROCKET PHARMACEUTICALS LEADERSHIP, ROCKET PHARMACEUTICALS, INC.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Isabel Carmona, J.D., as chief human resources officer and senior vice president.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Isabel Carmona, J.D., as chief human resources officer and senior vice president.
  • I am delighted to welcome Isabel to Rocket and believe her rich experience leading organizational growth at life science companies will be a tremendous asset as we continue our exciting growth trajectory, said Gaurav Shah, M.D., chief executive officer of Rocket Pharma.
  • Prior to joining Rocket, Ms. Carmona was chief human resources officer of Ichnos Sciences.
  • Prior to that, she served in positions of increasing leadership in the global operations and human resources teams at Teva Pharmaceuticals and Shire Pharmaceuticals.

Rocket Pharmaceuticals Announces $26.4 Million Private Placement

Retrieved on: 
Monday, August 30, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Company, Private Securities Litigation Reform Act, Investment, Securities Act of 1933, Genetic disorder, U.S. Securities and Exchange Commission, LVV, AAV, SEC, Research, Bone marrow failure, IMO, RTW, PKD, Osteopetrosis, Marketing, Fanconi anemia, LP, Medication, NASDAQ, Prospectus, Safety, Pyruvate kinase deficiency, Patient, Sale, FA, Lists of diseases, Annual report, COVID-19, Heart failure, Danon disease, Pharmaceutical industry

The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions.
  • Rocket expects to use the net proceeds from the private placement to continue to advance and expand its pipeline of product candidates, for research and development expenses and for working capital.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that aim to correct the root cause of complex and rare childhood disorders.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Announces FDA Lifts Clinical Hold on Danon Disease Trial of RP-A501

Retrieved on: 
Monday, August 16, 2021
Other Health, Research, General Health, Pharmaceutical, Genetics, Clinical Trials, Science, Cardiology, Biotechnology, FDA, Other Science, Health, Tissue, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASDAQ, Bone marrow failure, Danon disease, Adult, Male, Genetic disorder, FA, Company, Risk, Food, Private Securities Litigation Reform Act, Lists of diseases, Death, COVID-19, Program, LVV, SEC, Pyruvate kinase deficiency, Autophagy, FDA, Safety management system, Gene expression, PKD, Fanconi anemia, Patient, Osteopetrosis, Lifting, Marketing, IMO, Medication, Heart failure, Glycogen, Safety, AAV, Cardiac muscle, Autophagosome, Prevalence, Pharmaceutical industry, Vaccine, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, ROCKET PHARMACEUTICALS, INC.

We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.

Key Points: 
  • We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.
  • We look forward to progressing this critical work on behalf of all Danon patients, said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Rockets Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.