| Date | Title | Description |
| 08.12.2025 | Arrowhead Hits the Mark
‘Important step’ for Arrowhead
Redemplo: Targeting a rare disease
Expanded Redemplo uses and markets
Sarepta cash infusion | - |
| 04.11.2025 | Stocks making the biggest moves premarket: Uber, Sarepta, Palantir, Norwegian Cruise and more | - |
| 30.10.2025 | Hansa Biopharma Reports Third Quarter and Interim Year to Date 2025 Financial Results | Hansa Biopharma Reports Third Quarter and Interim Year to Date 2025 Financial Results
Thu, Oct 30, 2025 07:00 CET Report this content
Imlifidase successfully met primary endpoint in pivotal US Phase 3 ConfIdeS trial in kidney transplantatio... |
| 25.08.2025 | Kennedy’s anti-vaccine strategy risks forcing shots off market, manufacturers warn | Kennedy has said changes to the U.S. vaccine system are needed because, he asserts without evidence, immunizations are linked to autism, neurotoxicity, allergies, and death. He is a leader of the “Make America Healthy Again” movement, an in... |
| 01.08.2025 | Hansa Biopharma announces supportive data from treatment with imlifidase prior to the administration of gene therapy for Duchenne muscular dystrophy | Hansa Biopharma announces supportive data from treatment with imlifidase prior to the administration of gene therapy for Duchenne muscular dystrophy
Fri, Aug 01, 2025 21:15 CET Report this content
Lund, Sweden, 1 August 2025. Hansa Biopharm... |
| 30.07.2025 | FDA vaccine chief leaving agency after less than 3 months | His contrarian approach appeared to match that of his boss FDA Commissioner Marty Makary, who repeatedly praised Prasad’s work and intellect.
But in recent weeks Prasad became a target of right-wing activists, including Laura Loomer, who fl... |
| 30.07.2025 | Stocks making the biggest moves midday: Harley-Davidson, Wingstop, Sarepta Therapeutics and more | - |
| 29.07.2025 | Stocks making the biggest moves premarket: Novo Nordisk, Whirlpool, Sarepta Therapeutics and more | - |
| 29.07.2025 | Stocks making the biggest moves midday: Carrier, Johnson Controls, Chart Industries, Sarepta and more | - |
| 28.07.2025 | S&P 500 retreats from record, Dow falls 200 points with China talks in limbo and Fed decision ahead: Live updates | The S&P 500 closed lower on Tuesday as progress on trade talks with Beijing stalled and traders braced for the Federal Reserve’s rate decision.
The broad market index lost 0.30%, ending at 6,370.86. The Nasdaq Composite slipped 0.38%, s... |
| 21.07.2025 | FDA taps biotech industry veteran as RFK Jr.’s top drug regulator | The Food and Drug Administration said it has appointed former biotech executive George Tidmarsh as the agency’s top drug regulator.
Tidmarsh, an adjunct professor of pediatrics and neonatology at Stanford University’s School of Medicine, wi... |
| 20.07.2025 | S&P 500 posts first close above 6,300, Nasdaq hits a record ahead of big tech earnings: Live updates | The S&P 500 moved higher on Monday as optimism around earnings overshadowed any investor fears over the latest developments in trade.
The broad market index rose 0.14% and closed at 6,305.60 — marking the first time it ended a session a... |
| 19.07.2025 | Sarepta Refused FDA’s Request to Halt Elevidys Shipments | - |
| 18.07.2025 | Sarepta Says Another Patient Died After Receiving Gene Therapy | - |
| 18.07.2025 | Sarepta shares plunge 40% as future of its gene therapy appears at risk | Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk.
Sarepta has reported three patient deaths related to its gene therapies.
The Food and Drug Administration will reque... |
| 17.07.2025 | Sarepta Slashes Workforce After Patient Deaths on Gene Therapy | - |
| 17.07.2025 | Dow closes more than 100 points lower after report says Trump seeks at least 15% tariff on EU imports: Live updates | The Dow Jones Industrial Average slid Friday after President Donald Trump reportedly pushed for greater tariffs on the European Union.
The 30-stock Dow fell 142.30 points, or 0.32%, settling at 44,342.19. The S&P 500 lost 0.01% after hi... |
| 17.07.2025 | Hansa Biopharma Reports Second Quarter and Interim January-June 2025 Financial Results | Hansa Biopharma Reports Second Quarter and Interim January-June 2025 Financial Results
Thu, Jul 17, 2025 07:00 CET Report this content
Hansa secures directed cash share issue of approximately 232 MSEK/US $24.3M and restructures NovaQuest de... |
| 16.06.2025 | Second patient death reported with gene therapy for muscular dystrophy | Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product’s use.
Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness ... |
| 27.03.2025 | Leading Scientists and Advocates Unite for 'OHio, What a Night' to Accelerate Charcot-Marie-Tooth Disease Research | CMT Research Foundation Hosts Exclusive Event Spotlighting Groundbreaking Advances in CMT Treatment in Columbus COLUMBUS, OH, UNITED STATES, March 27, 2025 /EINPresswire.com/ -- The CMT Research Foundation is bringing together world-renowne... |
| 17.02.2025 | Sarepta Therapeutics Closes $600M in Senior Secured Revolving Credit Facility | Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a Cambridge, MA-based precision genetic medicine for rare diseases company, raised $600M in senior secured revolving facility with a bank syndicate.
JPMorgan Chase Bank, N.A. served as Administrativ... |
| 20.06.2024 | FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy | SILVER SPRING, Md., June 20, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ... |
| 20.06.2024 | PPMD Applauds FDA Decision to Expand ELEVIDYS Label for Ages Four Years and Older | WASHINGTON, June 20, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), applauds the U.S. Food and Drug Administration (FDA) for its decis... |
| 17.04.2024 | Parent Project Muscular Dystrophy Celebrates 30th Anniversary with Launch of PPMD Together Meeting Series in Cincinnati, Ohio | PPMD Together: Cincinnati Marks Three Decades of PPMD's Impact and Mayor's Proclamation
Honors Founding President & CEO Pat Furlong's Dedication to the Duchenne Community
CINCINNATI, April 17, 2024 /PRNewswire/ -- Parent Project Muscula... |
| 02.04.2024 | Hansa Biopharma Expects Strong First Quarter 2024 Sales Performance | Hansa Biopharma Expects Strong First Quarter 2024 Sales Performance
Tue, Apr 02, 2024 07:00 CET Report this content
Company expects revenue of SEK 54m including product sales of SEK 48m - an increase of 10% versus the prior quarter and a 23... |
| 22.03.2024 | FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease | Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each of these therapies only treats certain ... |
| 21.03.2024 | Hansa Biopharma publishes 2023 Annual and Sustainability Reports | Hansa Biopharma publishes 2023 Annual and Sustainability Reports
Thu, Mar 21, 2024 07:00 CET Report this content
Company delivered significant progress across commercial and R&D priorities
Lund, Sweden, March 21, 2024. Hansa Biopharma A... |
| 11.03.2024 | Global $111+ Billion Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis 2024-2034 - Rapid Growth in Exon-Skipping Therapies Reflects Precision Medical Interventions Fueled by R&D | - |
| 09.01.2022 | Hansa Biopharma provides business update including certain key financials | Hansa Biopharma provides business update including certain key financials Sun, Jan 09, 2022 08:00 CET
Commercial launch and market access efforts in Europe progressing as planned; New multiregional commercialization partnership with Medison... |
| 09.11.2021 | Patient registries: A catalyst for developing new therapies for rare diseases | Patient registries are powerful tools for collecting information about a variety of conditions or therapies. Observational patient registries may be designed to collect foundational information about a condition or to collect information ab... |
| 03.11.2021 | Sarepta Therapeutics Announces Third Quarter 2021 Financial Results and Recent Corporate Developments | Net product sales for the third quarter of 2021 reached $166.9 million, a 37% increase over the same quarter of prior yearIn light of its continued over-performance, Sarepta raises its full-year product revenue guidance by $40 million to be... |
| 27.08.2021 | Sarepta Therapeutics : Measurements Used in Clinical Trials for Duchenne Muscular Dystrophy – an Overview | Sarepta is working hard to develop precision genetic medicines for patients with rare diseases who often have limited or no treatment options, including Duchenne. Clinical trials are critical for understanding the safety and effectiveness o... |
| 07.11.2019 | Software companies continue the winning streak on Deloitte’s Fast 500 list | On the 25th anniversary of Deloitte’s Fast 500 list, software companies are still eating the world but their diet has expanded to include roadside assistance, personal loans, and online video creation. The top company–UiPath–is all about te... |
| 01.07.2019 | Pfizer gene therapy shows early efficacy in Duchenne muscular dystrophy, but data raise questions | In addition to assessing the safety and tolerability of the gene therapy, the study also looked at a secondary efficacy endpoint of mini-dystrophin distribution within muscle fibers. Dystrophin is a protein that is mainly concentrated in th... |
| 22.03.2016 | FDA urged to approve Sarepta DMD drug by dozens of medical experts | More than three dozen medical experts are urging the Food and Drug Administration to approve a Sarepta Therapeutics medicine for combating Duchenne muscular dystrophy, a rare disease that causes muscles in boys to stop working and eventuall... |
| 12.08.2015 | Checkmate nets $20M to develop drug that boosts checkpoint inhibitor efficacy | Checkmate Pharmaceuticals operates on the idea that checkpoint inhibitor therapies would be much more effective if they were combined with an agent that could stimulate a person’s immune response. So, on top of the new financing, it’s also ... |
| 14.04.2015 | Duchenne Muscular Dystrophy market to shoot up 100-fold in five years | “These mutation-specific drugs are expected to have a high price point due to their novelty, efficacy and orphan drug status, and will contribute 85.6% to the DMD treatment arena by 2019,” GlobalData analyst Nikhilesh Sanyal said in a state... |
| - | Checkmate nets $20M to develop drug that boosts checkpoint inhibitor efficacy | The name of the company’s pretty pithy: Checkmate Pharmaceuticals, so-called because its drug in development serves as a “mate” to checkpoint inhibitors, enhancing their efficacy.
The Cambridge company just launched with a $20 million Serie... |
| - | Pfizer gene therapy shows early efficacy in Duchenne muscular dystrophy, but data raise questions | Data presented at a medical conference last Friday provided a peek at the potential efficacy of a gene therapy under development by Pfizer for a rare genetic disorder that causes muscle degeneration.
The New York-based drugmaker on presente... |
| - | Duchenne Muscular Dystrophy market to shoot up 100-fold in five years | As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, the global market for the degenerative disease is expected to expand at an incredible rate – from $8.2 million in 2014 to $990 million in 2019,... |
