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Aug 14, 2026
Keros Therapeutics plans a phase II DMD trial of rinvatercept in Q1 2026 and regulatory talks for ALS in H2 2026. Cash stands at $383 million with runway into H1 2028.
Jun 20, 2026
India convened a national conference on rare diseases, Irish lawmakers debated Skyclarys access, and Malta unveiled its first rare disease strategy. New measures include expanded Centres of Excellence, ₹50 lakh assistance, and givinostat reimbursement.
Jul 15, 2026
Researchers at MD Anderson developed an extracellular vesicle-based platform that delivers full-length DMD mRNA, restoring dystrophin production and improving muscle function in preclinical Duchenne muscular dystrophy models. The study, published in Nature Biomedical Engineering, showed reduced side effects compared with viral gene therapies.
Aug 07, 2026
Biotech companies posted Q2 2026 results: REGENXBIO's Duchenne trial met its primary endpoint, Compass Pathways' COMP360 NDA submission is on track for Q4, Puma raised full-year guidance on NERLYNX demand, and Evotec cut its 2026 outlook.
Aug 07, 2026
BioMarin reported Q2 2026 revenues of $990 million, up 20% Y/Y, and raised guidance. It filed an sNDA for VOXZOGO in hypochondroplasia and advanced BMN 333, BMN 820, and BMN 351.
Jun 24, 2026
A new Italian White Paper reveals women with rare diseases wait two years longer than men for a diagnosis, while Georgia expands state-funded care for rare neuromuscular conditions to adults. The report also highlights regional disparities and economic burdens on patients and carers.
Jun 15, 2026
FDA reinstates top vaccines regulator ten days after abrupt departure, drawing criticism from public health experts and far-right commentators. Departure was tied to Duchenne muscular dystrophy treatment and COVID-19 vaccine controversies.
Jul 10, 2026
Phase III data show deramiocel slowed upper limb decline by 54% in Duchenne muscular dystrophy. The FDA is re-evaluating the BLA with HOPE-3 results, with a submission of the clinical study report expected in February 2026.
Jun 12, 2026
The FDA granted Rare Pediatric Disease designation to Atossa Therapeutics' (Z)-endoxifen for treating Duchenne Muscular Dystrophy. The designation may qualify the drug for a valuable Priority Review Voucher upon approval. Atossa reported a net loss of $34.8 million for 2025 as it advances clinical development.
Jun 05, 2026
Servier will acquire Edgewise Therapeutics' muscular dystrophy business, including the drug sevasemten, for up to $2.65 billion. Sevasemten recently faced an FDA rejection for accelerated approval in Becker muscular dystrophy but is advancing to a Phase 3 trial in 2026. The drug has received multiple FDA and EMA designations for both Becker and Duchenne muscular dystrophy.
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.
