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May 14, 2026
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Apr 23, 2026
Sarepta Therapeutics said it will announce first quarter 2026 financial results. The event will be webcast on its investor relations website, with a replay archived for one year.
Apr 18, 2026
India's rare disease treatment funding fell to Rs 32.73 crore in 2025-26 from Rs 82.87 crore a year earlier, while several Centres of Excellence reported unspent balances. In Madhya Pradesh, concerns were also raised over the absence of dedicated rare disease centres in most state-run medical colleges.
Apr 16, 2026
Mesoblast received FDA clearance for a registrational DMD trial of remestemcel-L-rknd, while Satellos began dosing boys in the Phase 2 BASECAMP study of SAT-3247. The studies are enrolling children ages 5-9 and 7-9, respectively.
Apr 08, 2026
Duchenne muscular dystrophy care is shifting toward broader multidisciplinary management and closer attention to early pathology, biomarkers, and treatment timing. Clinicians described an expanding treatment landscape and identified neurology, cardiology, pulmonary, and endocrinology as core priorities.
Mar 29, 2026
Wearable technologies are transforming clinical trials and healthcare with continuous monitoring capabilities, though clinical validation remains limited. While ECG features provide useful data for cardiac monitoring, many wearable metrics lack clinical accuracy and can cause patient anxiety. The technology shows promise for activity tracking and specific medical applications but requires cautious interpretation.
Mar 25, 2026
Diamyd Medical anticipates March 2026 interim results from its Phase 3 diabetes trial that could support a BLA pathway. Capricor Therapeutics awaits an August 2026 FDA decision on its Duchenne muscular dystrophy treatment BLA. Bicara Therapeutics prepares for Phase 3 study initiation of its oncology candidate toward future BLA submission.
Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 13, 2026
The FDA has lifted a Complete Response Letter and resumed review of Capricor Therapeutics' Biologics License Application for deramiocel, setting a PDUFA date of August 22nd. The company ended 2025 with $318M in cash.
Mar 12, 2026
Scientists have developed two breakthrough approaches to dramatically enhance gene editing and mRNA therapy delivery: a simple amino acid supplement that increases CRISPR efficiency to nearly 90 percent, and a self-replicating CRISPR system that spreads between cells like a virus.
Mar 12, 2026
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Mar 09, 2026
Precision BioSciences receives FDA Fast Track designation for PBGENE-DMD, a gene editing therapy for Duchenne muscular dystrophy designed to treat patients with mutations in exons 45-55.
Mar 09, 2026
Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.
Mar 07, 2026
The FDA has denied or discouraged at least eight drug applications in the past year, including treatments for rare diseases, raising concerns about regulatory consistency and the future of experimental therapies.
Mar 06, 2026
Dyne Therapeutics is preparing a U.S. Accelerated Approval submission for z-rostudirsen in Duchenne muscular dystrophy and gearing up for a Phase 3 trial of z-basivarsen in myotonic dystrophy, while scaling operations to support potential commercialization.
Mar 06, 2026
The FDA has placed a partial hold on U.S. enrollment in a Phase 2 trial of del-desiran for myotonic dystrophy type 1, citing preclinical mouse data showing blood pressure drops not seen in humans, following a serious adverse event in earlier testing.
Mar 01, 2026
Artificial intelligence is revolutionizing rare disease diagnosis, cutting diagnostic timelines from years to weeks. Rare Disease Day on February 28 highlights challenges faced by over 300 million people worldwide living with more than 7,000 distinct rare conditions.
Feb 28, 2026
Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.
Feb 28, 2026
Insmed reported total revenues of $606.4 million for 2025, with ARIKAYCE growing 19% annually. The FDA granted orphan drug designation to treprostinil palmitil for pulmonary arterial hypertension treatment in January 2026.
Feb 26, 2026
Edgewise Therapeutics and IDEAYA Biosciences released annual financial reports detailing clinical trial progress and strategic initiatives. Both companies are advancing multiple clinical-stage programs while managing significant operating expenses.
