All Rare diseases articles – Page 2
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NewsGene therapy trial facilitates major vision improvement
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
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NewsEuropean Commission licenses first treatment for acanthamoeba keratitis
Following its European approval, the small molecule treatment is set to become the standard of care for patients with the ultra-rare disease.
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NewsFDA approves innovative engineered cell therapy
The accelerated approval by the US Food and Drug Administration (FDA) authorises the first engineered cell therapy for a solid tumour in the US.
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ArticleThe route to enhanced gene therapies
In this article, Roger Palframan, Head of US Research at UCB, delves into the potential of gene therapy and which modalities have the most promise, what the industry should prioritise to advance the field, as well as what is needed to develop the workforce.
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NewsCHMP 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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ArticleDrug approval roundup – May/June 2024
This article highlights some of the key regulatory approvals granted in May and June in the EU and US so far this year, including for oligonucleotide and biologic therapies.
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ArticleWhy patient-centricity is essential to accelerate rare disease clinical research
There is an urgent need for novel treatment options for people living with rare diseases, but carrying out clinical research in this field is notoriously difficult. As Clinical Trials Day approaches, Cezary Statuch, Head of Medical Affairs International and Japan at Alexion Pharmaceuticals, highlights how adopting patient-centric approaches can help ...
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NewsCHMP meeting highlights: April 2024
In its April meeting, the EMA’s human medicines committee recommended eight new medicines, including treatments for cancer and autoimmune diseases.
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NewsBenefits of 3D printing for personalised medicine formulation
A study has demonstrated how 3D printing can be used to manufacture tailored, safe, and effective treatments for patients with rare diseases via pharmaceutical compounding.
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NewsNovartis releases new data for innovative rare kidney disease treatment
Data from the pre-specified interim analysis of Fabhalta® (iptacopan) indicate a statistically significant reduction in proteinuria reduction.
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NewsmRNA therapy could provide intracellular protein replacement for rare disease
With no treatments approved for propionic acidaemia, Moderna’s LNP-encapsulated mRNA therapy could offer a new therapeutic option for patients, new data suggests.
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NewsCHMP meeting highlights: March 2024
At the Committee for Medicinal Products for Human Use (CHMP)’s March meeting, twelve medicines received positive opinions for marketing authorisations, including a new antibiotic and a novel oral medicine for a rare blood disorder.
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NewsAstraZeneca agrees novel $1 billion endocrine acquisition
As part of the proposed acquisition, Alexion (AstraZeneca Rare Disease) will add a therapeutic to its pipeline that has potential to “shift the treatment paradigm” in hypoparathyroidism.
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ArticleMaking ATMPs a reality for rare disease patients
Advanced therapy medicinal products (ATMPs) will be among the first to undergo joint clinical assessments at EU level in 2025 and these will form the basis for national value assessments and pricing negotiations. Here, Paolo Morgese from the Alliance for Regenerative Medicine discusses how methodologies for joint clinical assessments (JCAs) ...
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NewsCHMP meeting highlights: February 2024
Ten new medicines, including a new oligonucleotide therapy for ALS, were recommended for approval at the Committee for Medicinal Products for Human Use (CHMP)’s recent meeting.
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NewsCHMP 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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NewsSanofi bets $1.7b on potential best-in-class rare disease therapy
Under its newly agreed acquisition, Sanofi is to acquire Inhibrx, Inc., adding INBRX-101 to its portfolio, a therapy that could improve the treatment options available for alpha-1 antitrypsin deficiency (AATD).
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NewsAstraZeneca to advance haematology cell therapy
The new acquisition grants AstraZeneca rights to a new CAR-T cell therapy with a differentiated manufacturing process that could provide a potential best-in-class blood cancer treatment.
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VideomRNA therapies: a new frontier
Nicolas Chornet, Senior Vice President of International Manufacturing at Moderna, speaks to EPR about the future of mRNA therapies and offers an update on the company’s international expansion plans.
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NewsMSD medicine accepted for use in Scotland
Scotland is the first European country to accept the HIF-2α inhibitor WELIREG® (belzutifan) for eligible adults with von Hippel-Lindau (VHL) disease.
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