Tag: rare disease

LEXINGTON, Mass. and AMSTERDAM, June 25, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the closing of its previously announced underwritten public offering of 5,686,813 ordinary shares at a public offering price of $45.50 per share, which includes 741,758 ordinary shares issued pursuant to the exercise in full by the underwriters of their option to purchase additional ordinary shares in the offering. The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, were approximately $259 million. All securities in the offering were sold by uniQure.

 ABO-101 is an investigational gene editing therapy designed to address the underlying disease biology of PH1 by reducing hepatic oxalate production

RIYADH, Saudi Arabia, June 12, 2026 (GLOBE NEWSWIRE) -- King Faisal Specialist Hospital & Research Centre (KFSH) will use its participation at HLTH Europe 2026, taking place from June 15 to 18 in Amsterdam, to highlight the strategic value of tertiary care datasets as a foundation for advancing artificial intelligence, precision medicine, and clinical innovation in highly specialized healthcare.

MILAN, Italy, June 10, 2026 – Italfarmaco S.p.A. today announced that Health Canada has accepted for review the New Drug Submission (NDS) for givinostat (Duvyzat^®) for the treatment of Duchenne muscular dystrophy (DMD). Health Canada has granted givinostat Priority Review status, a designation reserved for drugs that may provide a significant improvement in the benefit-risk profile over existing therapies.

“Families affected by Duchenne muscular dystrophy in Canada currently have limited access to treatment options that can slow functional decline,” said Dr. Jean K. Mah, a Canadian investigator for the EPIDYS and OLE studies and Director of the Pediatric Neuromuscular Program at the Alberta Children’s Hospital in Calgary, Alberta. “As a clinician treating DMD patients, I am encouraged by this positive step in the Canadian regulatory progress and givinostat’s therapeutic potential demonstrated in the EPIDYS Phase 3 study."

An estimated 1000 boys in Canada are living with DMD, one of the most severe and common forms of childhood muscular dystrophy.¹ As part of its commitment to the Canadian DMD community, Italfarmaco will establish a local Canadian affiliate to lead ongoing discussions with health authorities throughout the regulatory review process and to support patients and their families as well as healthcare providers. Subject to Health Canada’s review, marketing authorisation could be granted before the end of 2026.

“Duchenne muscular dystrophy places an enormous burden on boys and their families, and we understand the urgency of bringing new treatment options to communities where significant unmet need remains,” said Francesco Di Marco, Chief Executive Officer of Italfarmaco Group. “Health Canada’s acceptance of the New Drug Submission for givinostat with Priority Review is an encouraging step forward. As we expand our presence in Canada, we are committed to working with clinicians, patient organisations, and other stakeholders to support the Duchenne community and help advance access for appropriate patients, if approved.”

The submission builds on many years of clinical research, including contributions from Canadian patients, families, investigators, and three clinical trial sites in Canada. It is supported by data from the EPIDYS Phase 3 multicentre, randomized, double-blind, placebo-controlled trial (NCT02851797) evaluating givinostat in ambulant boys with DMD aged six and older. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment in patients treated with givinostat twice daily, in addition to corticosteroids, compared to placebo and corticosteroids. During the study, the most common treatment-related adverse events (frequency ≥1/10 boys) associated with givinostat were decreased platelet count/thrombocytopenia, increased blood triglyceride/hypertriglyceridemia, diarrhoea and abdominal pain; none of the severe or serious adverse events were treatment-related or resulted in study withdrawal. givinostat tolerability was managed with appropriate monitoring and dose adjustments. No other safety concerns were observed.² Patients from the EPIDYS study remain under observation in a long-term study assessing tolerability and efficacy, with follow-up extending up to eleven years (NCT03373968).

Givinostat has received regulatory approvals in multiple regions, including the US, the UK, the EU, and the UAE for the treatment of DMD. Additional regulatory submissions are ongoing in other geographies, reflecting Italfarmaco’s commitment to enabling access for as many individuals living with DMD as possible.


About Duchenne Muscular Dystrophy

COPENHAGEN, Denmark, June 08, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the latest data from its Endocrinology Rare Disease programs in hypoparathyroidism, achondroplasia, and pediatric growth hormone deficiency will be shared in two oral presentations and three posters at ENDO 2026, the annual meeting of the Endocrine Society being held in Chicago from June 13-16, 2026.

European Commission approval expands the indication of lomitapide in the European Union (EU) to include children 5 years of age and older with HoFH, an ultra-rare genetic disorder affecting LDL-cholesterol levels