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CHMP meeting highlights – June 2024
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines, including positive opinions for treatments for rare disorders.
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Duchenne muscular dystrophy (DMD)
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AAV gene therapy demonstrates positive activity in Duchenne muscular dystrophy
Initial trial results show the gene therapy enabled up to 85 percent of muscle fibres expressing microdystrophin in boys with Duchenne muscular dystrophy eight weeks post-injection.
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Clinical trials for rare diseases
In this interview, Mindy Leffler, Managing Director of Qualitative Research and Psychometrics at Emmes Endpoint Solutions, discusses the nuances of designing clinical trials for rare diseases including specific challenges related to traditional endpoints.
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CHMP meeting highlights: January 2024
In its first meeting of 2024, the CHMP recommended a generic medicine for schizophrenia and refused a marketing authorisation (MA) for geographic atrophy.
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MHRA approves ‘safer’ medicine alternative for rare disease
A new drug has been approved in the UK as a safer alternative to corticosteroids to treat Duchenne muscular dystrophy (DMD).
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CHMP’s September 2023 meeting highlights
In its September 2023 meeting, the EMA’s human medicines committee (CHMP) recommended nine medicines for approval and the extension of therapeutic indications for 11 medicines.
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Gene therapy approved for Duchenne muscular dystrophy
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
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mRNA therapeutics: a limitless revolution in medicine
Carsten Rudolph and Christian Plank of Ethris, an mRNA therapeutics and vaccine‑focused German biotechnology company, discuss the current state of the art in the field, including delivery systems and administration of mRNA drug candidates to prevent or treat a plethora of diseases.
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Challenges in downstream purification of advanced therapies
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
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Dystrogen begins trial for Duchenne muscular dystrophy cell therapy
The first patient has been infused in the Phase I pilot study of DT-DEC01, a chimeric cell therapy for Duchenne muscular dystrophy.
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Five marketing strategies for novel therapeutics
Long before the advent of Coronavirus, next-generation therapies were making headlines, but the successful development of COVID-19 vaccines, including mRNA vaccines, has now shone a spotlight on next-generation therapies. This offers a boost to the long-term growth projections for the advanced therapy medicinal products (ATMPs) sector. But although ATMPs hold…
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Babies with SMA able to swallow with Evrysdi® treatment
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
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First UK patient enrolled in Duchenne muscular dystrophy gene therapy trial
The Phase III trial will evaluate the safety and efficacy of Pfizer’s PF-06939926 gene therapy in 99 paediatric Duchenne muscular dystrophy patients across 15 countries.
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Treatment for rare Duchenne muscular dystrophy mutation approved by FDA
The FDA conditionally approved Sarepta’s Amondys 45 (casimersen) after interim Phase III results indicated the treatment is reasonably likely to be of clinical benefit.
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Positive results in Duchenne muscular dystrophy (DMD) trial
Interim trial results suggest vamorolone may offer a superior alternative to long-term treatment with high-dose glucocorticoids in young Duchenne muscular dystrophy (DMD) patients.
