RARECast
RARECast
Categorieën: Zakelijk
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522 - A Small Molecule Therapy to Regenerate Muscle in People with DMDThu, 18 Apr 2024
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521 - Forging a Faster Path for Gene TherapiesThu, 11 Apr 2024
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520 - Determining the Value of Rare Disease TherapiesThu, 04 Apr 2024
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519 - Buying and Building a Gene Therapy PresenceThu, 28 Mar 2024
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518 - A Clinical Trial Failure Derails a Promising TechnologyThu, 21 Mar 2024
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517 - Helping People with Undiagnosed Rare Diseases Find AnswersThu, 14 Mar 2024
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516 - Speeding and Scaling the Development of Genome Editing TherapiesThu, 07 Mar 2024
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515 - Getting a Next-Generation Genome Editing Therapy for Sickle Cell Disease Back on TrackThu, 29 Feb 2024
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514 - Trying to Break a Leg in a WheelchairThu, 22 Feb 2024
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513 - A Drug Developer that Makes Pediatric Cancers a PriorityThu, 15 Feb 2024
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512 - Harnessing the Body’s Natural RNA Machinery to Treat DiseasesThu, 08 Feb 2024
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511 - Targeting the Leading Cause of Death in Friedreich’s Ataxia with a Gene TherapyThu, 01 Feb 2024
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510 - How Inhaled mRNA May Help Rare Disease Patients Breathe EasierThu, 25 Jan 2024
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509 - Changing What’s Possible with Cell and Gene TherapiesThu, 18 Jan 2024
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508 - Why Flying with a Wheelchair Is a Civil Rights IssueThu, 11 Jan 2024
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507 - Mapping Spheres of InfluenceThu, 04 Jan 2024
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506 - Bringing Precision to the Treatment of Rare CancersThu, 28 Dec 2023
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505 - The Challenges of Developing a Therapy for Pregnant Women with A Rare ConditionThu, 21 Dec 2023
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504 - How One Drug Developer Is Addressing Health InequitiesThu, 14 Dec 2023
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503 - Accelerating the Development of Genetic Medicines with AIThu, 07 Dec 2023
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502 - Leveraging AI to Address the Neuropsychiatric Conditions Associated with a Rare DiseaseThu, 30 Nov 2023
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501 - In Pursuit of a Treatment for His Son, A Lawyer Becomes a Biotech ExecutiveWed, 22 Nov 2023
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500 - In Push for Treatment, a Patient Organization Becomes a Trial SponsorThu, 16 Nov 2023
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499 - Delayed Diagnosis of Rare Diseases Takes Staggering Economic TollThu, 09 Nov 2023
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498 - A Cell Therapy to Control Heart-Damaging Inflammation in DuchenneThu, 02 Nov 2023
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497 - Stimulating the Growth of Muscle Mass in People with Neuromuscular DiseasesThu, 26 Oct 2023
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496 - Turning Grief into ActionThu, 19 Oct 2023
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495 - A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair HearingThu, 12 Oct 2023
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494 - How the Woolly Mammoth Beat a Path to Better Gene TherapiesThu, 05 Oct 2023
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493 - A Genetic Counselor’s Journey into Patient AdvocacyThu, 28 Sep 2023
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492 - A Company in the Fold of Rare DiseaseThu, 21 Sep 2023
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491 - Disabling Antibodies that Drive Rare, Immunological ConditionsThu, 14 Sep 2023
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490 - Keeping the Sentinels of the Brain VigilantThu, 07 Sep 2023
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489 - Considering the Unfinished Work in Cystic FibrosisThu, 31 Aug 2023
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488 - How to Advocate for Yourself as You Battle CancerThu, 24 Aug 2023
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487 - Targeting Neuroinflammation to Treat ALSThu, 17 Aug 2023
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486 - Leveraging Community Partnerships to Address a Rare Disease Behind a Medical Mystery in New MexicoThu, 10 Aug 2023
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485 - Accelerating Gene Editing Therapies for Rare, Neurological ConditionsThu, 03 Aug 2023
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484 - Inhibiting Electrical Activity in Rare, Seizure DisordersThu, 27 Jul 2023
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483 - Improving the Function and Usability of Clothing for People with DisabilitiesThu, 20 Jul 2023
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482 - Treating Cardiopulmonary Disease with Inhaled Targeted TherapiesThu, 13 Jul 2023
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481 - A Patient-Driven Registry Focused on Health-Related Quality of Life DataThu, 29 Jun 2023
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480 - An Ally for the Undiagnosed EmergesThu, 29 Jun 2023
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479 - Help for Rare Disease Patients That’s Just a Click AwayThu, 22 Jun 2023
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478 - Diagnosing Autism with a Single Strand of HairThu, 15 Jun 2023
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477 - Bringing Regenerative Medicine to a Rare Bone Condition in ChildrenThu, 08 Jun 2023
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476 - Bringing Precision to CRISPR-Based Genome EditingThu, 01 Jun 2023
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475 - Capturing Patients’ Experience in Their Daily LivesThu, 25 May 2023
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474 - From One to ManyThu, 18 May 2023
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473 - A Nasal Spray to Curb Excessive Eating in Prader-WilliThu, 11 May 2023
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