Krystal Biotech announced that, on May 15, 2026, the United Kingdom MHRA granted marketing authorization to Vyjuvek (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis… read more.
Denali Therapeutics Inc. announced the FDA has granted accelerated approval for Avlayah (tividenofusp alfa-eknm), the first FDA-approved biologic specifically designed to cross the blood-brain barrier and reach the… read more.
A collaboration between Karolinska Institutet, Karolinska University Hospital, and SciLifeLab has integrated whole genome sequencing into routine diagnostic investigations for rare diseases at Karolinska University Hospital. To date,… read more.
Ascendis Pharma announced that Yuviwel (navepegritide; developed as TransCon CNP) was granted orphan drug exclusivity by the FDA and that Yuviwel is now commercially available in the United… read more.
In short-skulled (brachycephalic) dogs, a very flat face, collapsing nostrils, and a plump physique are associated with difficulty breathing, with Pekingeses and Japanese Chins at especially high risk,… read more.
PTC Therapeutics Inc. announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following… read more.
Zevra Therapeutics announced the presentation of four posters highlighting positive new data on Miplyffa (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at the 22nd Annual… read more.
Health Canada has issued a Notice of Compliance with conditions (NOC/c) for Voxzogo (vosoritide for injection) a modified recombinant human C-type natriuretic peptide 39 (CNP-39) for the treatment… read more.
Camurus announced that the FDA has accepted for review the company’s resubmission of the New Drug Application (NDA) for Oclaiz (CAM 2029), octreotide extended-release injection, for the treatment… read more.
Arrowhead Pharmaceuticals, Inc. announced that the Chinese National Medical Products Administration (NMPA) has approved Redemplo (plozasiran) for the reduction of triglyceride levels in adult patients with familial chylomicronemia… read more.
Abeona Therapeutics Inc. announced the first patient treatment with FDA-approved Zevaskyn (prademagene zamikeracel), a first-of-its-kind, autologous gene therapy for treating wounds in adult and pediatric patients with recessive… read more.
A test using genetics to predict who is at the highest risk of developing type 1 diabetes could one day be applied to largescale health studies, to identify… read more.
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