Rare disease Archives

Best in Biotech 28 Feb 2025

Rare Disease Day: seven drugs awaiting approval in 2025

As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in 2025.

February 28, 2025 - 10 minutesmins - By Roohi Mariam Peter

Best in Biotech 20 Feb 2025

3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder

Discover three promising approaches being investigated to treat Hunter syndrome, a rare genetic disorder affecting children.

February 20, 2025 - 10 minutesmins - By Willow Shah-Neville

News and Trends 2 Oct 2024

Niemann-Pick disease: FDA approves first two drugs in the space of a week for rare disorder

Find out all about the first two FDA drug approvals for the rare genetic disorder Niemann-Pick disease type C.

October 2, 2024 - 7 minutesmins - By Willow Shah-Neville

Podcast 7 Jun 2024

Hope for tomorrow: Developing treatments for rare pediatric diseases

On this week’s podcast we are discussing novel treatments of rare pediatric diseases with Dan Williams, CEO, executive director of Synaptix Bio.

June 7, 2024

Podcast 24 May 2024

How biotech is tackling myasthenia gravis

To talk about the disease, how it is being treated, and what is in the pipeline for future treatments, we had a conversation with Dana Vigier, head of Central Eastern and Northern Europe at Alexion.

May 24, 2024

In Depth 15 May 2024

Drugs for IgA nephropathy on the rise in the clinic

As we observed IgA Nephropathy Awareness Day on May 14th, let us take a look at ongoing clinical trials for drugs that aim to make patient lives better.

May 15, 2024 - 9 minutesmins - By Roohi Mariam Peter

In Depth 3 May 2024

Can emerging new treatments lead the way to cure Duchenne muscular dystrophy?

Delve into the new treatment approaches to Duchenne muscular dystrophy, a challenging disease for those suffering from it, and research.

May 3, 2024

Rare disease clinical trials: how can complex innovative designs help overcome the challenges?

Complex innovative designs: tackling rare disease clinical trial challenges

Unlock the power of complex innovative designs to tackle rare disease clinical trial challenges efficiently.

April 23, 2024 - 7 minutesmins - By Ute Boronowsky

In Depth 14 Mar 2024

Rett syndrome: advances in gene therapy brings hope

Explore the latest advancements in treating Rett syndrome, a rare genetic disorder, including groundbreaking gene therapies and drug trials.

March 14, 2024 - 8 minutesmins - By Roohi Mariam Peter

Podcast 6 Oct 2023

Beyond Biotech podcast 66 – treating rare diseases with tRNA

Compared to the smallest mRNA molecule, which is 300 nucleotides long, the largest transfer RNAs (tRNA) is less than a third of the size at 76 nucleotides. Scientists at Alltrna are harnessing the unique biology of tRNAs to engineer a single tRNA medicine that could treat thousands of rare diseases that share the same genetic […]

October 6, 2023

Opinion 20 Sep 2023

The next frontier in genomic technologies for rare diseases

By Neil Ward, VP and General Manager at PacBio EMEA It’s been 40 years since the passage of the Orphan Drug Act, which catalyzed the development of hundreds of therapies for rare diseases, with more than half approved in the last decade. More recently in 2022, the U.K. government launched a Rare Diseases Action Plan […]

September 20, 2023 - 7 minutesmins - By External Contributor

News and Trends 14 Jul 2023

Life saved: AI discovers existing drug works for rare disease

A patient’s life was saved after researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata, and Every Cure, used artificial intelligence to use an existing drug to treat idiopathic multicentric Castleman disease (iMCD). Every Cure and its partners are developing medical databases and AI algorithms to unlock new uses for existing medicines across […]

July 14, 2023 - 4 minutesmins - By Jim Cornall

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Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Rare disease

Rare Disease Day: seven drugs awaiting approval in 2025

3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder

Niemann-Pick disease: FDA approves first two drugs in the space of a week for rare disorder

Hope for tomorrow: Developing treatments for rare pediatric diseases

How biotech is tackling myasthenia gravis

Drugs for IgA nephropathy on the rise in the clinic

Can emerging new treatments lead the way to cure Duchenne muscular dystrophy?

Complex innovative designs: tackling rare disease clinical trial challenges

Rett syndrome: advances in gene therapy brings hope

Beyond Biotech podcast 66 – treating rare diseases with tRNA

The next frontier in genomic technologies for rare diseases

Life saved: AI discovers existing drug works for rare disease

Subscribe to our newsletter to get the latest biotech news!

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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