Yasmin Sheikh

Regulating therapies for rare diseases – recent approvals

This article summarises some of the recent notable drug approvals…

8 August 2024 | By Catherine Eckford (European Pharmaceutical Review)

This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.

Website development by e-Motive Media Limited.

Cookie Settings

This website uses cookies to improve your experience while you navigate through the website. Out of these cookies, the cookies that are categorised as ”Necessary” are stored on your browser as they are as essential for the working of basic functionalities of the website. For our other types of cookies “Advertising & Targeting”, “Analytics” and “Performance”, these help us analyse and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these different types of cookies. But opting out of some of these cookies may have an effect on your browsing experience. You can adjust the available sliders to ‘Enabled’ or ‘Disabled’, then click ‘Save and Accept’. View our Cookie Policy page.

Necessary

Always Enabled

Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.

Cookie	Description
cookielawinfo-checkbox-advertising-targeting	The cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Advertising & Targeting".
cookielawinfo-checkbox-analytics	This cookie is set by GDPR Cookie Consent WordPress Plugin. The cookie is used to remember the user consent for the cookies under the category "Analytics".
cookielawinfo-checkbox-necessary	This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Necessary".
cookielawinfo-checkbox-performance	This cookie is set by GDPR Cookie Consent WordPress Plugin. The cookie is used to remember the user consent for the cookies under the category "Performance".
PHPSESSID	This cookie is native to PHP applications. The cookie is used to store and identify a users' unique session ID for the purpose of managing user session on the website. The cookie is a session cookies and is deleted when all the browser windows are closed.
viewed_cookie_policy	The cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. It does not store any personal data.
zmember_logged	This session cookie is served by our membership/subscription system and controls whether you are able to see content which is only available to logged in users.

Performance

Performance cookies are includes cookies that deliver enhanced functionalities of the website, such as caching. These cookies do not store any personal information.

Cookie	Description
cf_ob_info	This cookie is set by Cloudflare content delivery network and, in conjunction with the cookie 'cf_use_ob', is used to determine whether it should continue serving “Always Online” until the cookie expires.
cf_use_ob	This cookie is set by Cloudflare content delivery network and is used to determine whether it should continue serving “Always Online” until the cookie expires.
free_subscription_only	This session cookie is served by our membership/subscription system and controls which types of content you are able to access.
ls_smartpush	This cookie is set by Litespeed Server and allows the server to store settings to help improve performance of the site.
one_signal_sdk_db	This cookie is set by OneSignal push notifications and is used for storing user preferences in connection with their notification permission status.
YSC	This cookie is set by Youtube and is used to track the views of embedded videos.

Analytics

Analytics cookies collect information about your use of the content, and in combination with previously collected information, are used to measure, understand, and report on your usage of this website.

Cookie	Description
bcookie	This cookie is set by LinkedIn. The purpose of the cookie is to enable LinkedIn functionalities on the page.
GPS	This cookie is set by YouTube and registers a unique ID for tracking users based on their geographical location
lang	This cookie is set by LinkedIn and is used to store the language preferences of a user to serve up content in that stored language the next time user visit the website.
lidc	This cookie is set by LinkedIn and used for routing.
lissc	This cookie is set by LinkedIn share Buttons and ad tags.
vuid	We embed videos from our official Vimeo channel. When you press play, Vimeo will drop third party cookies to enable the video to play and to see how long a viewer has watched the video. This cookie does not track individuals.
wow.anonymousId	This cookie is set by Spotler and tracks an anonymous visitor ID.
wow.schedule	This cookie is set by Spotler and enables it to track the Load Balance Session Queue.
wow.session	This cookie is set by Spotler to track the Internet Information Services (IIS) session state.
wow.utmvalues	This cookie is set by Spotler and stores the UTM values for the session. UTM values are specific text strings that are appended to URLs that allow Communigator to track the URLs and the UTM values when they get clicked on.
_ga	This cookie is set by Google Analytics and is used to calculate visitor, session, campaign data and keep track of site usage for the site's analytics report. It stores information anonymously and assign a randomly generated number to identify unique visitors.
_gat	This cookies is set by Google Universal Analytics to throttle the request rate to limit the collection of data on high traffic sites.
_gid	This cookie is set by Google Analytics and is used to store information of how visitors use a website and helps in creating an analytics report of how the website is doing. The data collected including the number visitors, the source where they have come from, and the pages visited in an anonymous form.

Advertising & Targeting

Advertising and targeting cookies help us provide our visitors with relevant ads and marketing campaigns.

Cookie	Description
advanced_ads_browser_width	This cookie is set by Advanced Ads and measures the browser width.
advanced_ads_page_impressions	This cookie is set by Advanced Ads and measures the number of previous page impressions.
advanced_ads_pro_server_info	This cookie is set by Advanced Ads and sets geo-location, user role and user capabilities. It is used by cache busting in Advanced Ads Pro when the appropriate visitor conditions are used.
advanced_ads_pro_visitor_referrer	This cookie is set by Advanced Ads and sets the referrer URL.
bscookie	This cookie is a browser ID cookie set by LinkedIn share Buttons and ad tags.
IDE	This cookie is set by Google DoubleClick and stores information about how the user uses the website and any other advertisement before visiting the website. This is used to present users with ads that are relevant to them according to the user profile.
li_sugr	This cookie is set by LinkedIn and is used for tracking.
UserMatchHistory	This cookie is set by Linkedin and is used to track visitors on multiple websites, in order to present relevant advertisement based on the visitor's preferences.
VISITOR_INFO1_LIVE	This cookie is set by YouTube. Used to track the information of the embedded YouTube videos on a website.

Recommended

Yasmin Sheikh

Regulating therapies for rare diseases – recent approvals